Scholars@Duke
Richard Stanley Bedlack

Richard Stanley Bedlack

Stewart, Hughes and Wendt Distinguished Professor
Neurology, Neuromuscular Disease
bedla001@mc.duke.edu
Duke Box 3333, Durham, NC 27710
932 Morreene Rd ROOM234, Durham, NC

Selected Publications

The Scientific and Therapeutic Rationale for Off-Label Treatments in Amyotrophic Lateral Sclerosis.

Journal Article Ann Neurol · November 6, 2024 There are no dramatically effective pharmacological treatments for most patients with amyotrophic lateral sclerosis, a complex disease with multiple underlying mechanisms, such as neuroinflammation, oxidative stress, mitochondrial dysfunction, microbiome a ... Full text Link to item Cite

ALSUntangled #76: Wahls protocol.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · October 9, 2024 The Wahls diet is a modified Paleolithic diet that emphasizes dark green leafy vegetables, colorful fruits, high-quality animal proteins, and omega-3 polyunsaturated fatty acids, while limiting grains, legumes, dairy products, sugar, and processed foods co ... Full text Link to item Cite

Genetic Associations With an Amyotrophic Lateral Sclerosis Reversal Phenotype.

Journal Article Neurology · August 27, 2024 BACKGROUND AND OBJECTIVES: The term "ALS Reversal" describes patients who initially meet diagnostic criteria for amyotrophic lateral sclerosis (ALS) or had clinical features most consistent with progressive muscular atrophy (PMA) but subsequently demonstra ... Full text Link to item Cite

ALSUntangled #75: Portable neuromodulation stimulator therapy.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2024 Spurred by patient interest, ALSUntangled herein examines the potential of the Portable Neuromodulation Stimulator (PoNS™) in treating amyotrophic lateral sclerosis (ALS). The PoNS™ device, FDA-approved for the treatment of gait deficits in adult patients ... Full text Link to item Cite

Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.

Journal Article Muscle Nerve · August 2024 INTRODUCTION/AIMS: Expanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share ... Full text Link to item Cite

Rare variant analyses validate known ALS genes in a multi-ethnic population and identifies ANTXR2 as a candidate in PLS.

Journal Article BMC Genomics · June 29, 2024 BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting over 300,000 people worldwide. It is characterized by the progressive decline of the nervous system that leads to the weakening of muscles which impacts physical funct ... Full text Link to item Cite

Evaluating emerging drugs in phase II & III for the treatment of amyotrophic lateral sclerosis.

Journal Article Expert Opin Emerg Drugs · June 2024 INTRODUCTION: Amyotrophic Lateral Sclerosis is a rapidly progressive motor neuron disorder causing severe disability and premature death. Owing to the advances in uncovering ALS pathophysiology, efficient clinical trial design and research advocacy program ... Full text Link to item Cite

Revisiting Glutamate Excitotoxicity in Amyotrophic Lateral Sclerosis and Age-Related Neurodegeneration.

Journal Article Int J Mol Sci · May 21, 2024 Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disorder. While there are five FDA-approved drugs for treating this disease, each has only modest benefits. To design new and more effective therapies for ALS, particularly for sporadic AL ... Full text Link to item Cite

ALSUntangled #72: Insulin.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2024 ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review insulin, which has at least one plausible mechanism for slowing ALS progression. However, pre-clinical studies are limited ... Full text Link to item Cite

ALSUntangled #73: Lion's Mane.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2024 Lion's Mane (Hericium erinaceus) has historically been used as traditional medicine in Asia and Europe for its potential benefits in fighting infection and cancer. It has gained interest in the neurodegenerative disease field because of its mechanisms of a ... Full text Link to item Cite

ALSUntangled #71: Nuedexta.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2024 Nuedexta is a combination of dextromethorphan hydrobromide and quinidine sulfate and was approved by the Food and Drug Administration (FDA) in 2010 to treat pseudobulbar affect (PBA). There have since been anecdotal case reports of bulbar function improvem ... Full text Link to item Cite

Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.

Journal Article Ann Neurol · September 2023 Platform trials allow efficient evaluation of multiple interventions for a specific disease. The HEALEY ALS Platform Trial is testing multiple investigational products in parallel and sequentially in persons with amyotrophic lateral sclerosis (ALS) with th ... Full text Link to item Cite

ALSUntangled #66: antimycobacterial antibiotics.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2023 Several infections have been associated with motor neuron diseases resembling ALS, including species of viruses, bacteria, and parasites. Mycobacterium avium subspecies paratuberculosis (MAP), most known for its probable etiologic association with Crohn's ... Full text Link to item Cite

ALSUntangled #67: rituximab.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2023 ALSUntangled reviews alternative and off-label treatments on behalf of people with ALS who ask about them. Here we review rituximab, a drug which specifically depletes B lymphocytes. We show a current lack of evidence for a role of these cells in ALS progr ... Full text Link to item Cite

ALSUntangled #68: ozone therapy.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2023 ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review ozone therapy. Ozone therapy has possible mechanisms for slowing ALS progression based on its antioxidant, anti-inflammato ... Full text Link to item Cite

ALSUntangled # 69: astaxanthin.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2023 ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti- ... Full text Link to item Cite

The amyotrophic lateral sclerosis-health index (ALS-HI): development and evaluation of a novel outcome measure.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2023 Objective: The identification of effective therapeutics for ALS necessitates valid and responsive outcome measures to track disease progression and therapeutic gain in clinical trial settings. The Amyotrophic Lateral Sclerosis-Health Index (ALS-HI) is a mu ... Full text Link to item Cite

Intercellular transmission of pathogenic proteins in ALS: Exploring the pathogenic wave.

Journal Article Neurobiol Dis · August 2023 In patients with amyotrophic lateral sclerosis (ALS), disease symptoms and pathology typically spread in a predictable spatiotemporal pattern beginning at a focal site of onset and progressing along defined neuroanatomical tracts. Like other neurodegenerat ... Full text Link to item Cite

ALSUntangled #70: caffeine.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · June 8, 2023 ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here, we review caffeine which has plausible mechanisms for slowing ALS progression. However, pre-clinical studies are contradictory, and ... Full text Link to item Cite

Clenbuterol Treatment Is Safe and Associated With Slowed Disease Progression in a Small Open-Label Trial in Patients With Amyotrophic Lateral Sclerosis.

Journal Article J Clin Neuromuscul Dis · June 1, 2023 OBJECTIVE: Clenbuterol, a beta-agonist, has plausible mechanisms for treating amyotrophic lateral sclerosis (ALS). In this highly inclusive open-label trial (NCT04245709), we aimed to study the safety and efficacy of clenbuterol in patients with ALS. METHO ... Full text Link to item Cite

ALSUntangled #65: glucocorticoid corticosteroids.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2023 ALSUntangled reviews alternative and off-label treatments for people with amyotrophic lateral sclerosis (PALS). Here we review glucocorticoids. Neuroinflammation plays a prominent role in amyotrophic lateral sclerosis (ALS) pathogenesis, so some hypothesiz ... Full text Link to item Cite

ALSUntangled #63: ketogenic diets.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2023 ALSUntangled reviews alternative and off label treatments with a goal of helping patients make more informed decisions about them. Here we review ketogenic diets. We shows that these have plausible mechanisms, including augmenting cellular energy balance a ... Full text Link to item Cite

Study of "ALS reversals": LifeTime environmental exposures (StARLiTE).

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2023 We previously reported on a series of patients diagnosed with ALS whom had an extraordinary course defined by substantial and sustained improvement in weakness and function. For this study, twenty-five of these "ALS Reversals" completed extensive environme ... Full text Link to item Cite

Patient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional study.

Journal Article EClinicalMedicine · January 2023 BACKGROUND: As novel therapeutic interventions are being developed and tested in the amyotrophic lateral sclerosis (ALS) population, there is a need to better understand the symptoms and issues that have the greatest impact on the lives of individuals with ... Full text Link to item Cite

An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis.

Journal Article Expert Rev Neurother · January 2023 INTRODUCTION: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, fatal neurodegenerative motor neuron disease. Despite the overwhelming need for effective therapeutics for ALS, riluzole and edaravone were the only two FDA-approved disease-modify ... Full text Link to item Cite

Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid Biomarkers: A Review.

Journal Article JAMA Neurol · December 1, 2022 IMPORTANCE: Clinical trial activity in amyotrophic lateral sclerosis (ALS) is dramatically increasing; as a result, trial modifications have been introduced to improve efficiency, outcome measures have been reassessed, and considerable discussion about the ... Full text Link to item Cite

Filtered Cerebrospinal Fluid From Patients With Amyotrophic Lateral Sclerosis Displays an Altered Proteome and Affects Motor Phenotype in a Mouse Model.

Journal Article Cureus · December 2022 INTRODUCTION: Cerebrospinal fluid (CSF) has been implicated in amyotrophic lateral sclerosis (ALS) due to its ability to spread inflammatory proteins throughout the nervous system. We hypothesized that filtration of the CSF could remove pathogenic proteins ... Full text Link to item Cite

ALSUntangled #64: butyrates.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · November 2022 ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review butyrate and its different chemical forms (butyrates). Butyrates have plausible mechanisms for slowing ALS progression and ... Full text Link to item Cite

Intronic NEFH variant is associated with reduced risk for sporadic ALS and later age of disease onset.

Journal Article Sci Rep · August 30, 2022 Neurofilament heavy (NEFH) is one of the critical proteins required for the formation of the neuronal cytoskeleton and polymorphisms in NEFH are reported as a rare cause of sporadic ALS (sALS). In the current study, a candidate tetranucleotide (TTTA) repea ... Full text Link to item Cite

ALSUntangled #62: vitamin C.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2022 Vitamin C is one of the most common supplements taken by people with ALS. As an antioxidant, it has a plausible mechanism for slowing disease progression and there are some flawed pre-clinical studies and case reports suggesting benefit. However, a small h ... Full text Link to item Cite

Phase 2B randomized controlled trial of NP001 in amyotrophic lateral sclerosis: Pre-specified and post hoc analyses.

Journal Article Muscle Nerve · July 2022 INTRODUCTION/AIMS: ALS is a heterogeneous disease that may be complicated or in part driven by inflammation. NP001, a regulator of macrophage activation, was associated with slowing disease progression in those with higher levels of the plasma inflammatory ... Full text Link to item Cite

ALSUntangled #60: light therapy.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2022 ALSUntangled reviews alternative and off-label treatments for people with ALS. Here we review light therapy. We show that it has theoretically plausible mechanisms, three flawed pre-clinical data, studies, and one incompletely documented case report suppor ... Full text Link to item Cite

ALSUntangled #61: melatonin.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2022 ALSUntangled reviews alternative and off-label treatments for people with amyotrophic lateral sclerosis (ALS). Here we review melatonin. We show that it has plausible mechanisms, some positive (and some negative) pre-clinical studies, two cases in which co ... Full text Link to item Cite

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.

Journal Article Ann Neurol · February 2022 Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, ... Full text Link to item Cite

Recruitment of Patients With Amyotrophic Lateral Sclerosis for Clinical Trials and Epidemiological Studies: Descriptive Study of the National ALS Registry's Research Notification Mechanism.

Journal Article J Med Internet Res · December 7, 2021 BACKGROUND: Researchers face challenges in patient recruitment, especially for rare, fatal diseases such as amyotrophic lateral sclerosis (ALS). These challenges include obtaining sufficient statistical power as well as meeting eligibility requirements suc ... Full text Link to item Cite

Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers

Journal Article Current Treatment Options in Neurology · December 1, 2021 Purpose of Review: People with fatal neuromuscular diseases such as ALS want to access investigational products. Trials are our preferred pathway for this, but most people with these diseases will not be able to participate due to restrictive inclusion cri ... Full text Cite

MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design.

Journal Article Neurodegener Dis Manag · December 2021 Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with motor neuron loss as a defining feature. Despite significant effort, therapeutic breakthroughs have been modest. MN-166 (ibudilast) has demonstrated neuroprotective action by various m ... Full text Link to item Cite

ALSUntangled 59: Tamoxifen.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · November 2021 Here we use the ALSUntangled methodology to review Tamoxifen as an ALS treatment. We show that it has plausible mechanisms, a positive preclinical study, a case report and 2 small trials suggesting benefits. We show that it appears reasonably safe, though ... Full text Link to item Cite

Continuing Non-Invasive Ventilation During Amyotrophic Lateral Sclerosis-Related Hospice Care Is Medically, Administratively, and Financially Feasible.

Journal Article Am J Hosp Palliat Care · October 2021 BACKGROUND: Amyotrophic Lateral Sclerosis (ALS) is a terminal neuromuscular disease with patients dying within 3-5 years of diagnosis. Most patients choose to forego invasive life sustaining measures. Timing of hospice referral can be challenging given the ... Full text Link to item Cite

Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial.

Journal Article Lancet Neurol · October 2021 BACKGROUND: There is an urgent unmet need for new therapies in amyotrophic lateral sclerosis. In a clinical study with healthy volunteers, levosimendan, a calcium sensitiser, was shown to improve neuromechanical efficiency and contractile function of the h ... Full text Link to item Cite

Pathophysiology and Treatment of Non-motor Dysfunction in Amyotrophic Lateral Sclerosis.

Journal Article CNS Drugs · May 2021 Amyotrophic lateral sclerosis is a progressive and fatal neurodegenerative disease typically presenting with bulbar or limb weakness. There is increasing evidence that amyotrophic lateral sclerosis is a multisystem disease with early and frequent impacts o ... Full text Link to item Cite

ALSUntangled 57: Vinpocetine.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2021 Full text Link to item Cite

Recruitment of Patients With Amyotrophic Lateral Sclerosis for Clinical Trials and Epidemiological Studies: Descriptive Study of the National ALS Registry’s Research Notification Mechanism (Preprint)

Journal Article · February 17, 2021 BACKGROUNDResearchers face challenges in patient recruitment, especially for rare, fatal diseases such as amyotrophic lateral sclerosis (ALS). These challenges include obtaining sufficien ... Full text Cite

Total serum immunoglobulin A in ALS.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2021 Amyotrophic lateral sclerosis (ALS) can have marked phenotypic variability. To date, no biomarker explains this variability. This study tested the hypothesis that immunoglobulin A (IgA) levels might help explain the variability seen in ALS clinical present ... Full text Link to item Cite

Engaging ALS patients and caregivers (the ALS research ambassadors) to help design the REFINE-ALS biomarker study.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2021 In the planning and design of the Radicava/Edaravone Findings in Biomarkers From Amyotrophic Lateral Sclerosis (REFINE-ALS) study, we sought to elicit feedback from patients with ALS and their caregivers to ensure that patient-centric issues would be addre ... Full text Link to item Cite

Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

Journal Article JAMA Neurol · February 1, 2021 IMPORTANCE: Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease of the motor nervous system. Clinical studies have demonstrated cortical and spinal motor neuron hyperexcitability using transcranial magnetic stimulation and thresh ... Full text Link to item Cite

Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype.

Journal Article Front Aging Neurosci · 2021 OBJECTIVE: There is a critical need to establish genetic markers that explain the complex phenotypes and pathogenicity of ALS. This study identified a polymorphism in the Stathmin-2 gene and investigated its association with sporadic ALS (sALS) disease ris ... Full text Link to item Cite

ALSUntangled 56: "ten red flags"-things to be wary of in alternative or off-label products.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · November 2020 Full text Link to item Cite

Understanding the needs of people with ALS: a national survey of patients and caregivers.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2020 Objective: Amyotrophic lateral sclerosis (ALS) has profound effects on people with ALS (PALS) and caregivers. There is a paucity of research detailing and comparing PALS and caregiver day-to-day perspectives of ALS. Methods: A survey developed collaborativ ... Full text Link to item Cite

Amyotrophic lateral sclerosis care and research in the United States during the COVID-19 pandemic: Challenges and opportunities.

Journal Article Muscle Nerve · August 2020 Coronavirus disease 2019 has created unprecedented challenges for amyotrophic lateral sclerosis (ALS) clinical care and research in the United States. Traditional evaluations for making an ALS diagnosis, measuring progression, and planning interventions re ... Full text Link to item Cite

Disease-modifying effects of an SCAF4 structural variant in a predominantly SOD1 ALS cohort.

Journal Article Neurol Genet · August 2020 OBJECTIVE: To test the hypothesis that rs573116164 will have disease-modifying effects in patients with superoxide dismutase 1 (SOD1) familial amyotrophic lateral sclerosis (fALS), we characterized rs573116164 within a cohort of 190 patients with fALS and ... Full text Link to item Cite

ALS clinical research learning institutes (ALS-CRLI): empowering people with ALS to be research ambassadors.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2020 Objective: Patient engagement in research is increasingly recognized as important across many countries and fields. In 2008, we conducted surveys that suggested a need for improved patient engagement in ALS research. We decided to create an ALS Clinical Re ... Full text Link to item Cite

Plasma creatinine and oxidative stress biomarkers in amyotrophic lateral sclerosis.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2020 Objective: To determine the associations between plasma creatinine (PCr), plasma uric acid (PUA), and urinary oxidative stress (OS) biomarkers with the ALSFRS-R at baseline and survival in a large epidemiological cohort study (ALS COSMOS) with a well-pheno ... Full text Link to item Cite

Development and Validation of the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS).

Journal Article JAMA Neurol · April 1, 2020 IMPORTANCE: A new outcome measure for overall disability level with improved responsiveness is needed for amyotrophic lateral sclerosis (ALS) clinical trials. OBJECTIVE: To describe the creation and development of a new self-reported ALS disability scale w ... Full text Link to item Cite

Association of a structural variant within the SQSTM1 gene with amyotrophic lateral sclerosis.

Journal Article Neurol Genet · April 2020 OBJECTIVE: As structural variations may underpin susceptibility to complex neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), the objective of this study was to investigate a structural variant (SV) within sequestosome 1 (SQSTM1). METH ... Full text Link to item Cite

Study protocol for a randomised, double-blind, placebo-controlled study evaluating the Efficacy of cannabis-based Medicine Extract in slowing the disease pRogression of Amyotrophic Lateral sclerosis or motor neurone Disease: the EMERALD trial.

Journal Article BMJ Open · November 11, 2019 INTRODUCTION: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder with no known cure and with an average life expectancy of 3-5 years post diagnosis. The use of complementary medicine such as medicinal cannabis in search for a potential tre ... Full text Link to item Cite

ALSUntangled 48: Perampanel (Fycompa).

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2019 Full text Link to item Cite

Lunasin does not slow ALS progression: results of an open-label, single-center, hybrid-virtual 12-month trial.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2019 Objective: Lunasin, a soy peptide that reportedly alters histone acetylation in vitro, was associated with a single ALS reversal in the media. Following an ALSUntangled report, we sought to determine whether Lunasin altered histone acetylation and improved ... Full text Link to item Cite

ALSUntangled No. 47: RT001.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2019 Full text Link to item Cite

ALSUntangled 46: penicillin G/hydrocortisone.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2019 Full text Link to item Cite

Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial.

Journal Article Muscle Nerve · February 2019 INTRODUCTION: Rasagiline is a monoamine oxidase B (MAO-B) inhibitor with possible neuroprotective effects in patients with amyotrophic lateral sclerosis (ALS). METHODS: We performed a randomized, double-blind, placebo-controlled trial of 80 ALS participant ... Full text Link to item Cite

Corrigendum to "Longitudinal Screening Detects Cognitive Stability and Behavioral Deterioration in ALS Patients".

Journal Article Behav Neurol · 2019 [This corrects the article DOI: 10.1155/2018/5969137.]. ... Full text Link to item Cite

ALSUntangled 44: curcumin.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · November 2018 Full text Link to item Cite

"ALS reversals": demographics, disease characteristics, treatments, and co-morbidities.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · November 2018 OBJECTIVE: To identify differences in demographics, disease characteristics, treatments, and co-morbidities between patients with "amyotrophic lateral sclerosis (ALS) reversals" and those with typically progressive ALS. METHODS: Cases of possible ALS rever ... Full text Link to item Cite

ALSUntangled 43: copper.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · August 2018 Full text Link to item Cite

Independent home use of a brain-computer interface by people with amyotrophic lateral sclerosis.

Journal Article Neurology · July 17, 2018 OBJECTIVE: To assess the reliability and usefulness of an EEG-based brain-computer interface (BCI) for patients with advanced amyotrophic lateral sclerosis (ALS) who used it independently at home for up to 18 months. METHODS: Of 42 patients consented, 39 ( ... Full text Link to item Cite

ALSUntangled 42: Elysium health's "basis".

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2018 Full text Link to item Cite

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene.

Journal Article Neuron · March 21, 2018 To identify novel genes associated with ALS, we undertook two lines of investigation. We carried out a genome-wide association study comparing 20,806 ALS cases and 59,804 controls. Independently, we performed a rare variant burden analysis comparing 1,138 ... Full text Link to item Cite

A spatial analysis of amyotrophic lateral sclerosis (ALS) cases in the United States and their proximity to multidisciplinary ALS clinics, 2013.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2018 BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease that typically results in death within 2-5 years of initial symptom onset. Multidisciplinary ALS clinics (MDCs) have been established to provide specialty care to people living ... Full text Link to item Cite

ALSUntangled 41: "Eric Is Winning".

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2018 Full text Link to item Cite

Longitudinal Screening Detects Cognitive Stability and Behavioral Deterioration in ALS Patients.

Journal Article Behav Neurol · 2018 OBJECTIVE: To evaluate longitudinal cognitive/behavioral change over 12 months in participants enrolled in the ALS Multicenter Cohort Study of Oxidative Stress (ALS COSMOS). METHODS: We analyzed data from 294 ALS participants, 134 of whom were studied seri ... Full text Link to item Cite

Perspective: Untangling the ALS X-Files.

Journal Article Nature · October 18, 2017 Full text Link to item Cite

ALSUntangled No. 37: Inosine.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2017 Full text Link to item Cite

ALSUntangled 38: L-serine.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2017 Full text Link to item Cite

ALSUntangled No. 36: Accilion.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · February 2017 Full text Link to item Cite

Retinal thinning in amyotrophic lateral sclerosis patients without ophthalmic disease.

Journal Article PLoS One · 2017 IMPORTANCE: Amyotrophic lateral sclerosis (ALS) is a fatal, rapidly progressive neurodegenerative disease that primarily affects motor neurons. Recently, three causative genes have been implicated in both ALS and glaucoma. However, it is still uncertain wh ... Full text Link to item Cite

Association Between Dietary Intake and Function in Amyotrophic Lateral Sclerosis.

Journal Article JAMA Neurol · December 1, 2016 IMPORTANCE: There is growing interest in the role of nutrition in the pathogenesis and progression of amyotrophic lateral sclerosis (ALS). OBJECTIVE: To evaluate the associations between nutrients, individually and in groups, and ALS function and respirato ... Full text Link to item Cite

How common are ALS plateaus and reversals?

Journal Article Neurology · March 1, 2016 OBJECTIVE: To determine the frequency of amyotrophic lateral sclerosis (ALS) plateaus and reversals in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. METHODS: We analyzed Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFR ... Full text Link to item Cite

Cognitive-behavioral screening reveals prevalent impairment in a large multicenter ALS cohort.

Journal Article Neurology · March 1, 2016 OBJECTIVES: To characterize the prevalence of cognitive and behavioral symptoms using a cognitive/behavioral screening battery in a large prospective multicenter study of amyotrophic lateral sclerosis (ALS). METHODS: Two hundred seventy-four patients with ... Full text Link to item Cite

Palliative Care Issues in Amyotrophic Lateral Sclerosis: An Evidenced-Based Review.

Journal Article Am J Hosp Palliat Care · February 2016 As palliative care physicians become increasingly involved in the care of patients with amyotrophic lateral sclerosis (ALS), they will be asked to provide guidance regarding the use of supplements, diet, exercise, and other common preventive medicine inter ... Full text Link to item Cite

ALSUntangled No. 35: Hyperbaric Oxygen Therapy.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · 2016 Full text Link to item Cite

ALSUntangled No. 34: GM604.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · 2016 Full text Link to item Cite

Complementary and Alternative Therapies in Amyotrophic Lateral Sclerosis.

Journal Article Neurol Clin · November 2015 Given the severity of their illness and lack of effective disease-modifying agents, it is not surprising that most patients with amyotrophic lateral sclerosis (ALS) consider trying complementary and alternative therapies. Some of the most commonly consider ... Full text Link to item Cite

Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS.

Journal Article Neurol Neuroimmunol Neuroinflamm · June 2015 OBJECTIVE: To assess the safety, tolerability, and preliminary efficacy of NP001, a novel immune regulator of inflammatory monocytes/macrophages, for slowing progression of amyotrophic lateral sclerosis (ALS). METHODS: This was a phase 2 randomized, double ... Full text Link to item Cite

Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways.

Journal Article Science · March 27, 2015 Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease with no effective treatment. We report the results of a moderate-scale sequencing study aimed at increasing the number of genes known to contribute to predisposition for ALS. We perf ... Full text Link to item Cite

ALSUntangled: introducing The Table of Evidence.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · March 2015 Full text Link to item Cite

Comprehensive rehabilitative care across the spectrum of amyotrophic lateral sclerosis.

Journal Article NeuroRehabilitation · 2015 BACKGROUND: Amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) is a neurodegenerative disease that results in progressive muscle weakness and wasting. There is no known cure and the disease is uniformly fatal. PURPOSE: This review discusses curren ... Full text Link to item Cite

ALSUntangled No. 29: MitoQ.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · 2015 Full text Link to item Cite

ALSUntangled No. 26: lunasin.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · December 2014 Full text Link to item Cite

ALS Multicenter Cohort Study of Oxidative Stress (ALS COSMOS): study methodology, recruitment, and baseline demographic and disease characteristics.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · June 2014 Abstract In a multicenter study of newly diagnosed ALS patients without a reported family history of ALS, we are prospectively investigating whether markers of oxidative stress (OS) are associated with disease progression. Methods utilize an extensive stru ... Full text Link to item Cite

SAPs: a different perspective.

Journal Article Am J Bioeth · 2014 Full text Link to item Cite

Infrastructure resources for clinical research in amyotrophic lateral sclerosis.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2013 Clinical trial networks, shared clinical databases, and human biospecimen repositories are examples of infrastructure resources aimed at enhancing and expediting clinical and/or patient oriented research to uncover the etiology and pathogenesis of amyotrop ... Full text Link to item Cite

Peer recommendations on how to improve clinical research, and Conference wrap-up.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2013 To promote clinical and patient oriented research, as part of the Second International ALS Conference in Tarrytown, NY, USA, seven pairs of clinicians and scientists were asked to lead discussions with meeting attendees on six major topics (one of which wa ... Full text Link to item Cite

ALS Untangled No. 20: the Deanna protocol.

Journal Article Amyotroph Lateral Scler Frontotemporal Degener · May 2013 Full text Link to item Cite

Creatine for amyotrophic lateral sclerosis/motor neuron disease.

Journal Article Cochrane Database Syst Rev · December 12, 2012 BACKGROUND: Creatine, a naturally-occurring nitrogenous organic acid involved in adenosine triphosphate (ATP) production, has been shown to increase survival in mouse models of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND).  ... Full text Link to item Cite

ALS untangled No. 17: "when ALS is lyme".

Journal Article Amyotroph Lateral Scler · September 2012 Full text Link to item Cite

Diaphragm pacing in amyotrophic lateral sclerosis: a literature review.

Journal Article Muscle Nerve · July 2012 Amyotrophic lateral sclerosis (ALS) remains a rapidly progressive fatal degenerative disease of motor neurons for which there are few interventions to slow disease progression or improve quality of life. A diaphragm pacing system was approved by the U.S. F ... Full text Link to item Cite

Pesticide exposure and amyotrophic lateral sclerosis.

Journal Article Neurotoxicology · June 2012 Our objectives were to summarize literature on the association of amyotrophic lateral sclerosis (ALS) with pesticides as a group and to evaluate associations of ALS with specific pesticides. We conducted a meta-analysis of published studies of ALS and pest ... Full text Link to item Cite

ALSUntangled No. 16: cannabis.

Journal Article Amyotroph Lateral Scler · June 2012 Full text Link to item Cite

ALSUntangled 15: coconut Oil.

Journal Article Amyotroph Lateral Scler · May 2012 Full text Link to item Cite

Stem cell tourism and Canadian family physicians.

Journal Article Can Fam Physician · April 2012 Link to item Cite

Compassionate use of stem cells for ALS: popovers and hot air.

Journal Article Amyotroph Lateral Scler · September 2011 Full text Link to item Cite

HDACs in skeletal muscle remodeling and neuromuscular disease.

Journal Article Handb Exp Pharmacol · 2011 Skeletal muscle is made of heterogeneous myofibers with different contractile and metabolic properties. The diverse functionality of myofibers enables skeletal muscle to carry out different tasks from maintaining body posture to performing active movements ... Full text Link to item Cite

Modifiable barriers to enrollment in American ALS research studies.

Journal Article Amyotroph Lateral Scler · December 2010 Enrollment in ALS research studies is surprisingly low. Here we report on two online patient surveys that help identify some of the reasons. These include failure to invite patients to enroll, especially patients who have already participated in prior stud ... Full text Link to item Cite

Amyotrophic lateral sclerosis: current practice and future treatments.

Journal Article Curr Opin Neurol · October 2010 PURPOSE OF REVIEW: Knowledge of amyotrophic lateral sclerosis, and in particular the care of patients with it, is evolving exponentially. More than 1700 articles with the phrase 'amyotrophic lateral sclerosis' have been published in the past 2 years; these ... Full text Link to item Cite

Roaring in the ears: patulous Eustachian tube in bulbar amyotrophic lateral sclerosis.

Journal Article Amyotroph Lateral Scler · August 2010 We report a 64-year-old female with predominantly bulbar amyotrophic lateral sclerosis who experienced 'roaring' in her ears. She was diagnosed with patulous Eustachian tube, which is presumed to occur secondary to bulbar weakness. The roaring adversely af ... Full text Link to item Cite

Creatine for amyotrophic lateral sclerosis/motor neuron disease.

Journal Article Cochrane Database Syst Rev · June 16, 2010 BACKGROUND: Creatine, a naturally-occurring nitrogenous organic acid involved in adenosine triphosphate (ATP) production, has been shown to increase survival in mouse models of amyotrophic lateral sclerosis (ALS). Results from human trials, however, have b ... Full text Link to item Cite

Time to diagnosis in the National Registry of Veterans with Amyotrophic Lateral Sclerosis.

Journal Article Amyotroph Lateral Scler · 2010 Our objective was to determine the interval from symptom onset to diagnosis, and to evaluate associated factors in a cohort of U.S. Veterans with motor neuron diseases. We retrospectively evaluated 1359 patients enrolled in the National Registry of Veteran ... Full text Link to item Cite

Lyme disease serology in amyotrophic lateral sclerosis.

Journal Article Muscle Nerve · October 2009 Lyme disease is sometimes part of the differential diagnosis for amyotrophic lateral sclerosis (ALS). Herein we report on 414 individuals with ALS at the Massachusetts General Hospital who underwent laboratory testing for Lyme disease. Twenty-four (5.8%) w ... Full text Link to item Cite

Open-label pilot trial of levetiracetam for cramps and spasticity in patients with motor neuron disease.

Journal Article Amyotroph Lateral Scler · August 2009 Cramps and spasticity impair quality of life and function in patients with motor neuron diseases, and there are no proven treatments for these problems. We conducted a pilot trial to determine if treatment with levetiracetam was associated with a reduction ... Full text Link to item Cite

Phase 2 study of sodium phenylbutyrate in ALS.

Journal Article Amyotroph Lateral Scler · April 2009 The objective of the study was to establish the safety and pharmacodynamics of escalating dosages of sodium phenylbutyrate (NaPB) in participants with ALS. Transcription dysregulation may play a role in the pathogenesis of ALS. Sodium phenylbutyrate, a his ... Full text Link to item Cite

Factors associated with survival in the National Registry of Veterans with ALS.

Journal Article Amyotroph Lateral Scler · 2009 The clinical course of patients with ALS is highly variable. While the median survival time from symptom onset is 2-4 years, there are reports of survival ranging from less than a year to more than 40 years. Such variability makes planning difficult for pa ... Full text Link to item Cite

Als update: Signs of progress, easons for hope

Journal Article CONTINUUM Lifelong Learning in Neurology · January 1, 2009 Amyotrophic lateral sclerosis (ALS) was first described 134 years ago. While still incurable, significant progress has been made in understanding the pathophysiology of the disease and its management. For example, it is now clear that ALS is not a single d ... Full text Cite

Scrutinizing enrollment in ALS clinical trials: room for improvement?

Journal Article Amyotroph Lateral Scler · October 2008 Enrollment in ALS trials has not been systematically studied. We surveyed the ALS Research Group (ALSRG) to learn their impressions of enrollment at ALS clinics across North America. We also reviewed completed ALS trials to determine an enrollment rate (su ... Full text Link to item Cite

Creatine monohydrate in ALS: effects on strength, fatigue, respiratory status and ALSFRS.

Journal Article Amyotroph Lateral Scler · October 2008 Our objective was to determine the effect of creatine monohydrate on disease progression in patients with amyotrophic lateral sclerosis (ALS). One hundred and seven patients with the diagnosis of probable or definite ALS, of less than five years duration f ... Full text Link to item Cite

A novel, efficient, randomized selection trial comparing combinations of drug therapy for ALS.

Journal Article Amyotroph Lateral Scler · August 2008 Combining agents with different mechanisms of action may be necessary for meaningful results in treating ALS. The combinations of minocycline-creatine and celecoxib-creatine have additive effects in the murine model. New trial designs are needed to efficie ... Full text Link to item Cite

Genes and Environmental Exposures in Veterans with Amyotrophic Lateral Sclerosis: the GENEVA study. Rationale, study design and demographic characteristics.

Journal Article Neuroepidemiology · 2008 Recent reports of a potentially increased risk of amyotrophic lateral sclerosis (ALS) for veterans deployed to the 1990-1991 Persian Gulf War prompted the Department of Veterans Affairs to establish a National Registry of Veterans with ALS, charged with th ... Full text Link to item Cite

The National Registry of Veterans with amyotrophic lateral sclerosis.

Journal Article Neuroepidemiology · 2008 BACKGROUND: The Department of Veterans Affairs (VA) Cooperative Studies Program has established a National Registry of Veterans with Amyotrophic Lateral Sclerosis (ALS). This article describes the objectives, methods, and sample involved in the registry. M ... Full text Link to item Cite

Amyotrophic lateral sclerosis: an emerging era of collaborative gene discovery.

Journal Article PLoS One · December 5, 2007 Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease (MND). It is currently incurable and treatment is largely limited to supportive care. Family history is associated with an increased risk of ALS, and many Mendelian causes ... Full text Link to item Cite

Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial.

Journal Article Lancet Neurol · December 2007 Featured Publication BACKGROUND: Minocycline has anti-apoptotic and anti-inflammatory effects in vitro, and extends survival in mouse models of some neurological conditions. Several trials are planned or are in progress to assess whether minocycline slows human neurodegenerati ... Full text Link to item Cite

Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis.

Journal Article Expert Opin Emerg Drugs · May 2007 Featured Publication It has been > 130 years since the first description of the upper and lower motor neuron disease called amyotropic lateral sclerosis (ALS). Sadly, there has been little change in the long interval over which this disease is diagnosed, or in its poor prognos ... Full text Link to item Cite

Working through the challenges of neuropathic pain

Journal Article Emergency Medicine · October 1, 2005 The authors provide a guide to differentiating neuropathic from nociceptive pain, determining the cause of the pain, and choosing among first-, second-, and third-line therapeutic agents to achieve maximum pain control with a minimum of adverse effects. ... Cite

Quantitative myasthenia gravis score: assessment of responsiveness and longitudinal validity.

Journal Article Neurology · June 14, 2005 We prospectively tested the quantitative myasthenia gravis score (QMG) for responsiveness and longitudinal construct validity in 53 patients with myasthenia gravis. Index of responsiveness was high. Longitudinal construct validity was confirmed by the corr ... Full text Link to item Cite

Does this patient have myasthenia gravis?

Journal Article JAMA · April 20, 2005 CONTEXT: Clinicians must be able to diagnose myasthenia gravis, since delays in establishing the diagnosis may put patients at risk for complications from this treatable disease. OBJECTIVE: To determine if items in the history and examination or results of ... Full text Link to item Cite

MNGIE neuropathy: five cases mimicking chronic inflammatory demyelinating polyneuropathy.

Journal Article Muscle Nerve · March 2004 We report five patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) who had demyelinating peripheral neuropathy. The MNGIE neuropathy had clinical and electrodiagnostic features typical of acquired, rather than inherited, etiologies. ... Full text Link to item Cite

Diagnosing and stabilizing acute neuropathies

Journal Article Emergency Medicine · January 1, 2004 Focusing on the exceptional peripheral nervous system disorders in which symptoms progress rapidly, the authors review the presenting characteristics of immune-mediated, toxic, infectious, and metabolic acute neuropathies and identify the critical interven ... Cite

APOE genotype is a risk factor for neuropathy severity in diabetic patients.

Journal Article Neurology · March 25, 2003 This cross-sectional study tested the hypothesis that APOE genotype is a risk factor for diabetic neuropathy severity. A model with age, duration of diabetes, and APOE genotype was found to predict (p = 0.0083) severity on the Neuropathy Impairment Score i ... Full text Link to item Cite

Limitations of inferences from observational databases in amyotrophic lateral sclerosis: all that glitters is not gold.

Journal Article Amyotroph Lateral Scler Other Motor Neuron Disord · September 2002 Data from three observational databases have suggested that survival in patients with ALS who take riluzole is far greater than that reported in randomized controlled studies. This editorial discusses why therapeutic efficacy cannot be inferred from observ ... Full text Link to item Cite

On the concept of myasthenic crisis.

Journal Article J Clin Neuromuscul Dis · September 2002 This article reviews controversies surrounding the concept of myasthenic crisis. Literature review and our own experience demonstrate that there is disagreement about how a crisis should be defined, what the implications of a crisis are, whether crises are ... Full text Link to item Cite

Apolipoprotein E and neuromuscular disease: a critical review of the literature.

Journal Article Arch Neurol · November 2000 Molecular mechanisms that alter the incidence and rate of neuromuscular disease progression are, in many cases, only partially understood. Several recent studies have asked whether apolipoprotein E (apoE for the protein, APOE for the gene) influences these ... Full text Link to item Cite

Hidden afterdischarges in slow channel congenital myasthenic syndrome.

Journal Article J Clin Neuromuscul Dis · June 2000 Afterdischarges in motor nerve stimulation studies help distinguish slow channel congenital myasthenic syndrome (SCCMS) from acquired myasthenia gravis (MG) We present a patient with fatigable weakness in whom afterdischarges were not initially apparent. A ... Full text Link to item Cite

How to handle myasthenic crisis. Essential steps in patient care.

Journal Article Postgrad Med · April 2000 Myasthenic crisis, or respiratory failure requiring intubation and mechanical ventilation, may be caused by infections, aspiration, physical and emotional stress, and changes in medication. Although no single factor determines the need for respiratory supp ... Full text Link to item Cite

Transient lumbosacral polyradiculopathy after prostatectomy: association with spinal stenosis.

Journal Article South Med J · August 1999 Mononeuropathies are common after pelvic surgery. They are usually the result of unnatural positioning during surgery or faulty restraining devices. Polyneuropathy in the postoperative setting is rare. We report two cases of polyradiculopathy after radical ... Full text Link to item Cite

Succinylcholine induced hyperkalemia and cardiac arrest death related to an EEG study.

Journal Article J Clin Neurophysiol · January 1999 Changes in EEGs during cardiac arrest have been described in detail by many authors; however, mortality because of an EEG has never been reported. The authors report the case of a patient who developed cardiac arrest causally related to administration of s ... Full text Link to item Cite

Fluorescent imaging of membrane potential components

Conference ANALYTICAL USE OF FLUORESCENT PROBES IN ONCOLOGY · January 1, 1996 Link to item Cite

Distinct electric potentials in soma and neurite membranes.

Journal Article Neuron · November 1994 Structurally similar voltage-dependent ion channels may behave differently in different locations along the surface of a neuron. A possible reason could be that channels experience nonuniform electrical potentials along the plasmalemma. Here, we map the el ... Full text Link to item Cite

Dual-wavelength ratiometric fluorescence measurement of the membrane dipole potential.

Journal Article Biophys J · July 1994 The electrostatic potentials associated with cell membranes include the transmembrane potential (delta psi), the surface potential (psi s), and the dipole potential (psi D). psi D, which originates from oriented dipoles at the surface of the membrane, rise ... Full text Link to item Cite

Localized membrane depolarizations and localized calcium influx during electric field-guided neurite growth.

Journal Article Neuron · September 1992 Our study explores the mechanisms behind neurite galvanotropism. Using phase, differential interference contrast and ratiometric fluorescence microscopy, we reveal four responses of N1E-115 mouse neuroblastoma cells to 0.1-1.0 mV/microns uniform DC electri ... Full text Link to item Cite