Scholars@Duke
Jeffrey Guptill

Jeffrey Guptill

Adjunct Associate Professor in the Department of Neurology
Neurology, Neuromuscular Disease
jeffrey.guptill@duke.edu
Duke Box 3403, Duke South Clinic, 1L, Durham, NC 27710
Box 3403 DUMC, Duke South Clinic, 1L, Durham, NC 27710
Manage Profile
Embed Profile Data

Selected Publications

Immune dysregulation in chronic inflammatory demyelinating polyneuropathy.

Journal Article J Neuroimmunol · June 15, 2024 OBJECTIVE: Chronic inflammatory demyelinating polyneuropathy (CIDP) is an autoimmune disorder of the peripheral nerves with an incompletely understood underlying pathophysiology. This investigation focused on defining B and T cell frequencies, T cell funct ... Full text Link to item Cite

Identifying a stable and generalizable factor structure of major depressive disorder across three large longitudinal cohorts.

Journal Article Psychiatry Res · March 2024 The Patient Health Questionnaire 9 (PHQ-9) is the current standard outpatient screening tool for measuring and tracking the nine symptoms of major depressive disorder (MDD). While the PHQ-9 was originally conceptualized as a unidimensional measure, it has ... Full text Link to item Cite

Intravenous Ganaxolone: Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability in Healthy Adults.

Journal Article Clin Pharmacol Drug Dev · March 2024 Ganaxolone, a neuroactive steroid anticonvulsant that modulates both synaptic and extrasynaptic γ-aminobutyric acid type A (GABAA ) receptors, is in development for treatment of status epilepticus (SE) and rare epileptic disorders, and has been approved in ... Full text Link to item Cite

Comparative effectiveness of azathioprine and mycophenolate mofetil for myasthenia gravis (PROMISE-MG): a prospective cohort study.

Journal Article Lancet Neurol · March 2024 BACKGROUND: Myasthenia gravis is an autoimmune disorder of the neuromuscular junction. Treatment typically includes symptomatic oral cholinesterase inhibitors, immunosuppression, and immunomodulation. In addition to corticosteroids, azathioprine and mycoph ... Full text Link to item Cite

Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study.

Journal Article Neurology · January 23, 2024 BACKGROUND AND OBJECTIVES: To evaluate in a phase 2 study the safety and efficacy of IV nipocalimab, a fully human, antineonatal Fc receptor monoclonal antibody, in patients with generalized myasthenia gravis (gMG). METHODS: Patients with gMG with inadequa ... Full text Link to item Cite

Addressing Outcome Measure Variability in Myasthenia Gravis Clinical Trials.

Journal Article Neurology · September 5, 2023 An increasing number of clinical trials are enrolling patients with myasthenia gravis (MG). A lack of standardization in the performance of outcome measures leads to confusion among site research teams and is a source of variability in clinical trial data. ... Full text Link to item Cite

Clinicoserological insights into patients with immune checkpoint inhibitor-induced myasthenia gravis.

Journal Article Ann Clin Transl Neurol · May 2023 To compare the immunopathology of immune checkpoint inhibitor-induced myasthenia gravis (ICI-MG) and idiopathic MG, we profiled the respective AChR autoantibody pathogenic properties. Of three ICI-MG patients with AChR autoantibodies, only one showed compl ... Full text Link to item Cite

The Duke Myasthenia Gravis Clinic Registry: II. Analysis of outcomes.

Conference Muscle Nerve · April 2023 INTRODUCTION/AIMS: The Duke Myasthenia Gravis (MG) Clinic Registry contains comprehensive physician-derived data on patients with MG seen in the Duke MG Clinic since 1980. The aim of this study was to report outcomes in patients seen in the clinic and trea ... Full text Link to item Cite

The economic burden of individuals living with generalized myasthenia gravis and facing social determinants of health challenges

Journal Article Frontiers in Public Health · January 1, 2023 Objective: Better understanding the impact of social determinants of health (SDOH) barriers from the patient perspective is crucial to improve holistic patient support in generalized myasthenia gravis (gMG), a rare autoimmune disorder with high disease and ... Full text Cite

Pharmacokinetics of Ceftazidime-Avibactam in Combination with Aztreonam (COMBINE) in a Phase 1, Open-Label Study of Healthy Adults.

Journal Article Antimicrob Agents Chemother · December 20, 2022 Scant pharmacokinetic (PK) data are available on ceftazidime-avibactam (CZA) and aztreonam (ATM) in combination, and it is unknown if CZA-ATM exacerbates alanine aminotransferase (ALT)/aspartate aminotransferase (AST) elevations relative to ATM alone. This ... Full text Link to item Cite

Safety of Ceftazidime-Avibactam in Combination with Aztreonam (COMBINE) in a Phase I, Open-Label Study in Healthy Adult Volunteers.

Journal Article Antimicrob Agents Chemother · December 20, 2022 This phase I study evaluated the safety of the optimal ceftazidime-avibactam (CZA) with aztreonam (ATM) regimens identified in hollow fiber infection models of MBL-producing Enterobacterales. Eligible healthy subjects aged 18 to 45 years were assigned to o ... Full text Link to item Cite

Effect of FcRn antagonism on protective antibodies and to vaccines in IgG-mediated autoimmune diseases pemphigus and generalised myasthenia gravis.

Journal Article Autoimmunity · December 2022 Antagonism of the neonatal Fc receptor (FcRn) by efgartigimod has been studied in several autoimmune diseases mediated by immunoglobulin G (IgG) as a therapeutic approach to remove pathogenic IgGs. Whereas reduction of pathogenic titres has demonstrated ef ... Full text Link to item Cite

Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.

Journal Article Muscle Nerve · July 2022 INTRODUCTION/AIMS: Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive muscular dystrophy without approved therapies. In this study we evaluated whether locally acting ACE-083 could safely increase muscle volume and improve functional out ... Full text Link to item Cite

The clinical need for clustered AChR cell-based assay testing of seronegative MG.

Journal Article J Neuroimmunol · June 15, 2022 Trial eligibility in myasthenia gravis (MG) remains largely dependent on a positive autoantibody serostatus. This significantly hinders seronegative MG (SNMG) patients from receiving potentially beneficial new treatments. In a subset of SNMG patients, acet ... Full text Link to item Cite

Classical Complement Pathway Inhibition in a "Human-On-A-Chip" Model of Autoimmune Demyelinating Neuropathies.

Journal Article Adv Ther (Weinh) · June 2022 Chronic autoimmune demyelinating neuropathies are a group of rare neuromuscular disorders with complex, poorly characterized etiology. Here we describe a phenotypic, human-on-a-chip (HoaC) electrical conduction model of two rare autoimmune demyelinating ne ... Full text Link to item Cite

First-in-Human Clinical Trial to Assess the Safety, Tolerability and Pharmacokinetics of Single Doses of NTM-1633, a Novel Mixture of Monoclonal Antibodies against Botulinum Toxin E.

Journal Article Antimicrob Agents Chemother · April 19, 2022 Botulism is a rare, life-threatening paralytic disease caused by botulinum neurotoxin (BoNT). Available treatments including an equine antitoxin and human immune globulin are given postexposure and challenging to produce and administer. NTM-1633 is an equi ... Full text Link to item Cite

Adverse Reactions in a Phase 1 Trial of the Anti-Malarial DM1157: An Example of Pharmacokinetic Modeling and Simulation Guiding Clinical Trial Decisions.

Journal Article Infect Dis Ther · April 2022 INTRODUCTION: There is an urgent need to develop new drugs to treat malaria due to increasing resistance to first-line therapeutics targeting the causative organism, Plasmodium falciparum (P. falciparum). One drug candidate is DM1157, a small molecule that ... Full text Link to item Cite

Perioperative Outcomes of Thymectomy in Myasthenia Gravis: A Thoracic Surgery Database Analysis.

Journal Article Ann Thorac Surg · March 2022 BACKGROUND: There is clinical equipoise regarding the perioperative and long-term outcomes of autoimmune myasthenia gravis (MG) patients undergoing open vs minimally invasive thymectomy, particularly for nonthymomatous MG. This analysis utilizes multicente ... Full text Link to item Cite

Immune dysregulation in chronic inflammatory demyelinating polyneuropathy.

Journal Article J Neuroimmunol · June 15, 2024 OBJECTIVE: Chronic inflammatory demyelinating polyneuropathy (CIDP) is an autoimmune disorder of the peripheral nerves with an incompletely understood underlying pathophysiology. This investigation focused on defining B and T cell frequencies, T cell funct ... Full text Link to item Cite

Identifying a stable and generalizable factor structure of major depressive disorder across three large longitudinal cohorts.

Journal Article Psychiatry Res · March 2024 The Patient Health Questionnaire 9 (PHQ-9) is the current standard outpatient screening tool for measuring and tracking the nine symptoms of major depressive disorder (MDD). While the PHQ-9 was originally conceptualized as a unidimensional measure, it has ... Full text Link to item Cite

Intravenous Ganaxolone: Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability in Healthy Adults.

Journal Article Clin Pharmacol Drug Dev · March 2024 Ganaxolone, a neuroactive steroid anticonvulsant that modulates both synaptic and extrasynaptic γ-aminobutyric acid type A (GABAA ) receptors, is in development for treatment of status epilepticus (SE) and rare epileptic disorders, and has been approved in ... Full text Link to item Cite

Comparative effectiveness of azathioprine and mycophenolate mofetil for myasthenia gravis (PROMISE-MG): a prospective cohort study.

Journal Article Lancet Neurol · March 2024 BACKGROUND: Myasthenia gravis is an autoimmune disorder of the neuromuscular junction. Treatment typically includes symptomatic oral cholinesterase inhibitors, immunosuppression, and immunomodulation. In addition to corticosteroids, azathioprine and mycoph ... Full text Link to item Cite

Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study.

Journal Article Neurology · January 23, 2024 BACKGROUND AND OBJECTIVES: To evaluate in a phase 2 study the safety and efficacy of IV nipocalimab, a fully human, antineonatal Fc receptor monoclonal antibody, in patients with generalized myasthenia gravis (gMG). METHODS: Patients with gMG with inadequa ... Full text Link to item Cite

Addressing Outcome Measure Variability in Myasthenia Gravis Clinical Trials.

Journal Article Neurology · September 5, 2023 An increasing number of clinical trials are enrolling patients with myasthenia gravis (MG). A lack of standardization in the performance of outcome measures leads to confusion among site research teams and is a source of variability in clinical trial data. ... Full text Link to item Cite

Clinicoserological insights into patients with immune checkpoint inhibitor-induced myasthenia gravis.

Journal Article Ann Clin Transl Neurol · May 2023 To compare the immunopathology of immune checkpoint inhibitor-induced myasthenia gravis (ICI-MG) and idiopathic MG, we profiled the respective AChR autoantibody pathogenic properties. Of three ICI-MG patients with AChR autoantibodies, only one showed compl ... Full text Link to item Cite

The Duke Myasthenia Gravis Clinic Registry: II. Analysis of outcomes.

Conference Muscle Nerve · April 2023 INTRODUCTION/AIMS: The Duke Myasthenia Gravis (MG) Clinic Registry contains comprehensive physician-derived data on patients with MG seen in the Duke MG Clinic since 1980. The aim of this study was to report outcomes in patients seen in the clinic and trea ... Full text Link to item Cite

The economic burden of individuals living with generalized myasthenia gravis and facing social determinants of health challenges

Journal Article Frontiers in Public Health · January 1, 2023 Objective: Better understanding the impact of social determinants of health (SDOH) barriers from the patient perspective is crucial to improve holistic patient support in generalized myasthenia gravis (gMG), a rare autoimmune disorder with high disease and ... Full text Cite

Pharmacokinetics of Ceftazidime-Avibactam in Combination with Aztreonam (COMBINE) in a Phase 1, Open-Label Study of Healthy Adults.

Journal Article Antimicrob Agents Chemother · December 20, 2022 Scant pharmacokinetic (PK) data are available on ceftazidime-avibactam (CZA) and aztreonam (ATM) in combination, and it is unknown if CZA-ATM exacerbates alanine aminotransferase (ALT)/aspartate aminotransferase (AST) elevations relative to ATM alone. This ... Full text Link to item Cite

Safety of Ceftazidime-Avibactam in Combination with Aztreonam (COMBINE) in a Phase I, Open-Label Study in Healthy Adult Volunteers.

Journal Article Antimicrob Agents Chemother · December 20, 2022 This phase I study evaluated the safety of the optimal ceftazidime-avibactam (CZA) with aztreonam (ATM) regimens identified in hollow fiber infection models of MBL-producing Enterobacterales. Eligible healthy subjects aged 18 to 45 years were assigned to o ... Full text Link to item Cite

Effect of FcRn antagonism on protective antibodies and to vaccines in IgG-mediated autoimmune diseases pemphigus and generalised myasthenia gravis.

Journal Article Autoimmunity · December 2022 Antagonism of the neonatal Fc receptor (FcRn) by efgartigimod has been studied in several autoimmune diseases mediated by immunoglobulin G (IgG) as a therapeutic approach to remove pathogenic IgGs. Whereas reduction of pathogenic titres has demonstrated ef ... Full text Link to item Cite

Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.

Journal Article Muscle Nerve · July 2022 INTRODUCTION/AIMS: Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive muscular dystrophy without approved therapies. In this study we evaluated whether locally acting ACE-083 could safely increase muscle volume and improve functional out ... Full text Link to item Cite

The clinical need for clustered AChR cell-based assay testing of seronegative MG.

Journal Article J Neuroimmunol · June 15, 2022 Trial eligibility in myasthenia gravis (MG) remains largely dependent on a positive autoantibody serostatus. This significantly hinders seronegative MG (SNMG) patients from receiving potentially beneficial new treatments. In a subset of SNMG patients, acet ... Full text Link to item Cite

Classical Complement Pathway Inhibition in a "Human-On-A-Chip" Model of Autoimmune Demyelinating Neuropathies.

Journal Article Adv Ther (Weinh) · June 2022 Chronic autoimmune demyelinating neuropathies are a group of rare neuromuscular disorders with complex, poorly characterized etiology. Here we describe a phenotypic, human-on-a-chip (HoaC) electrical conduction model of two rare autoimmune demyelinating ne ... Full text Link to item Cite

First-in-Human Clinical Trial to Assess the Safety, Tolerability and Pharmacokinetics of Single Doses of NTM-1633, a Novel Mixture of Monoclonal Antibodies against Botulinum Toxin E.

Journal Article Antimicrob Agents Chemother · April 19, 2022 Botulism is a rare, life-threatening paralytic disease caused by botulinum neurotoxin (BoNT). Available treatments including an equine antitoxin and human immune globulin are given postexposure and challenging to produce and administer. NTM-1633 is an equi ... Full text Link to item Cite

Adverse Reactions in a Phase 1 Trial of the Anti-Malarial DM1157: An Example of Pharmacokinetic Modeling and Simulation Guiding Clinical Trial Decisions.

Journal Article Infect Dis Ther · April 2022 INTRODUCTION: There is an urgent need to develop new drugs to treat malaria due to increasing resistance to first-line therapeutics targeting the causative organism, Plasmodium falciparum (P. falciparum). One drug candidate is DM1157, a small molecule that ... Full text Link to item Cite

Perioperative Outcomes of Thymectomy in Myasthenia Gravis: A Thoracic Surgery Database Analysis.

Journal Article Ann Thorac Surg · March 2022 BACKGROUND: There is clinical equipoise regarding the perioperative and long-term outcomes of autoimmune myasthenia gravis (MG) patients undergoing open vs minimally invasive thymectomy, particularly for nonthymomatous MG. This analysis utilizes multicente ... Full text Link to item Cite

Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders.

Journal Article Lancet Neurol · February 2022 Autoimmune neuromuscular junction disorders are rare. However, myasthenia gravis is being increasingly recognised in people older than 50 years. In the past 5-10 years, epidemiological studies worldwide suggest an incidence of acetylcholine receptor antibo ... Full text Link to item Cite

Identifying a patient-centered outcome measure for a comparative effectiveness treatment trial in myasthenia gravis.

Journal Article Muscle Nerve · January 2022 INTRODUCTION/AIMS: Data regarding the comparative effectiveness of myasthenia gravis (MG) treatments is not available. We used patient input to identify a patient-centered outcome measure (PCOM) for PROMISE-MG, a comparative effectiveness trial of MG treat ... Full text Link to item Cite

Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study.

Journal Article Muscle Nerve · December 2021 INTRODUCTION/AIMS: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the effica ... Full text Link to item Cite

Editorial: News and Views in the Management of Myasthenia Gravis

Journal Article Frontiers in Neurology · October 7, 2021 Full text Cite

Cellular changes in eculizumab early responders with generalized myasthenia gravis.

Journal Article Clin Immunol · October 2021 Eculizumab (ECU), a C5 complement inhibitor, is approved to treat acetylcholine receptor autoantibody positive generalized myasthenia gravis (AChR MG). The clinical effect of ECU relies on inhibition of the terminal complement complex; however, the effect ... Full text Link to item Cite

Management/Treatment of Lambert-Eaton Myasthenic Syndrome

Journal Article Current Treatment Options in Neurology · October 1, 2021 Purpose of review: This article reviews the current treatment strategies for patients with Lambert-Eaton myasthenic syndrome (LEMS) including potassium channel blockers, immunosuppressant therapies, and management of dysautonomia. Recent findings: Since th ... Full text Cite

Telemedicine visits in myasthenia gravis: Expert guidance and the Myasthenia Gravis Core Exam (MG-CE).

Journal Article Muscle Nerve · September 2021 INTRODUCTION/AIMS: Telemedicine may be particularly well-suited for myasthenia gravis (MG) due to the disorder's need for specialized care, its hallmark fluctuating muscle weakness, and the potential for increased risk of virus exposure among patients with ... Full text Link to item Cite

Consensus disease definitions for neurologic immune-related adverse events of immune checkpoint inhibitors.

Journal Article J Immunother Cancer · July 2021 Expanding the US Food and Drug Administration-approved indications for immune checkpoint inhibitors in patients with cancer has resulted in therapeutic success and immune-related adverse events (irAEs). Neurologic irAEs (irAE-Ns) have an incidence of 1%-12 ... Full text Link to item Cite

Safety, Tolerability, and Pharmacokinetics of NTM-1632, a Novel Mixture of Three Monoclonal Antibodies against Botulinum Toxin B.

Journal Article Antimicrob Agents Chemother · June 17, 2021 Botulism is a rare, life-threatening paralytic disease caused by Clostridium botulinum neurotoxin (BoNT). Available treatments, including an equine antitoxin and human immune globulin, are given postexposure and challenging to produce and administer. NTM-1 ... Full text Link to item Cite

Reduced plasmablast frequency is associated with seronegative myasthenia gravis.

Journal Article Muscle Nerve · April 2021 BACKGROUND: The immunopathology of autoimmune seronegative myasthenia gravis (SN MG) is poorly understood. Our objective was to determine immune profiles associated with a diagnosis of SN MG. METHODS: We performed high-dimensional flow cytometry on blood s ... Full text Link to item Cite

Normative dataset for plasma cytokines in healthy human adults.

Journal Article Data Brief · April 2021 We determined normative data for plasma cytokines established from a cohort of 126 carefully screened healthy adults aged 18 to 64 years. Participants were enrolled to ensure an even age and sex distribution and to include at least 30% non-Caucasians. Plas ... Full text Open Access Link to item Cite

Knowledge and perceptions of the COVID-19 pandemic among patients with myasthenia gravis.

Journal Article Muscle Nerve · March 2021 BACKGROUND: Patients with myasthenia gravis (MG) may be particularly vulnerable during the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic due to risk of worsening disease during infection, potential adverse impacts of coronavirus dis ... Full text Link to item Cite

Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial.

Journal Article Neurology · February 9, 2021 OBJECTIVE: To explore the clinical efficacy and safety of subcutaneous (SC) rozanolixizumab, an anti-neonatal Fc receptor humanized monoclonal antibody, in patients with generalized myasthenia gravis (gMG). METHODS: In this phase 2a, randomized, double-bli ... Full text Link to item Cite

First-in-Human Studies of MW01-6-189WH, a Brain-Penetrant, Antineuroinflammatory Small-Molecule Drug Candidate: Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Studies in Healthy Adult Volunteers.

Journal Article Clin Pharmacol Drug Dev · February 2021 MW01-6-189WH (MW189) is a novel central nervous system-penetrant small-molecule drug candidate that selectively attenuates stressor-induced proinflammatory cytokine overproduction and is efficacious in intracerebral hemorrhage and traumatic brain injury an ... Full text Link to item Cite

The Duke myasthenia gravis clinic registry: I. Description and demographics.

Journal Article Muscle Nerve · February 2021 INTRODUCTION: The Duke Myasthenia Gravis (MG) Clinic Registry is a disease-specific database containing physician-derived data from patients seen in the Duke MG Clinic since 1980. METHODS: Data from 1060 MG patients initially seen between 1980 and 2008 wer ... Full text Link to item Cite

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.

Journal Article Neurology · January 26, 2021 OBJECTIVE: To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifes ... Full text Open Access Link to item Cite

A chimeric hemagglutinin-based universal influenza virus vaccine approach induces broad and long-lasting immunity in a randomized, placebo-controlled phase I trial.

Journal Article Nat Med · January 2021 Seasonal influenza viruses constantly change through antigenic drift and the emergence of pandemic influenza viruses through antigenic shift is unpredictable. Conventional influenza virus vaccines induce strain-specific neutralizing antibodies against the ... Full text Link to item Cite

Imbalance in T follicular helper cells producing IL-17 promotes pro-inflammatory responses in MuSK antibody positive myasthenia gravis.

Journal Article J Neuroimmunol · August 15, 2020 A detailed understanding of the role of Tfh cells in MuSK-antibody positive myasthenia gravis (MuSK-MG) is lacking. We characterized phenotype and function of Tfh cells in MuSK-MG patients and controls. We found similar overall Tfh and follicular regulator ... Full text Link to item Cite

Consistent improvement with eculizumab across muscle groups in myasthenia gravis.

Journal Article Ann Clin Transl Neurol · August 2020 OBJECTIVE: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively ... Full text Open Access Link to item Cite

'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.

Journal Article J Neurol · July 2020 BACKGROUND: The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), ... Full text Open Access Link to item Cite

Inhibition of the transcription factor ROR-γ reduces pathogenic Th17 cells in acetylcholine receptor antibody positive myasthenia gravis.

Journal Article Exp Neurol · March 2020 IL-17 producing CD4 T cells (Th17) cells increase significantly with disease severity in myasthenia gravis (MG) patients. To suppress the generation of Th17 cells, we examined the effect of inhibiting retinoic acid receptor-related-orphan-receptor-C (RORγ) ... Full text Link to item Cite

Immunogenicity of chimeric haemagglutinin-based, universal influenza virus vaccine candidates: interim results of a randomised, placebo-controlled, phase 1 clinical trial.

Journal Article Lancet Infect Dis · January 2020 BACKGROUND: Influenza viruses cause substantial annual morbidity and mortality globally. Current vaccines protect against influenza only when well matched to the circulating strains. However, antigenic drift can cause considerable mismatches between vaccin ... Full text Link to item Cite

Clinical outcome measures following plasma exchange for MG exacerbation.

Journal Article Ann Clin Transl Neurol · October 2019 Our objective is to report longitudinal results of the MG-ADL, MG-Composite, MG-MMT, and MG-QoL15 in an open-label trial of therapeutic plasma exchange in myasthenia gravis. Ten MG patients experiencing exacerbation had assessments prior to, immediately fo ... Full text Link to item Cite

Validation of the triple timed up-and-go test in Lambert-Eaton myasthenia.

Journal Article Muscle Nerve · September 2019 INTRODUCTION: There are no validated, practical, and quantitative measures of disease severity in Lambert-Eaton myasthenia (LEM). METHODS: Data from the Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome (DAPPER) trial were analyzed ... Full text Link to item Cite

Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Journal Article Qual Life Res · August 2019 PURPOSE: To evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subsca ... Full text Open Access Link to item Cite

Correction to: Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Journal Article Qual Life Res · August 2019 The article "Eculizumab improves fatigue in refractory generalized myasthenia gravis", written by "Henning Andersen, Renato Mantegazza, Jing Jing Wang, Fanny O'Brien, Kaushik Patra, James F. Howard Jr. and The REGAIN Study Group" was originally published e ... Full text Open Access Link to item Cite

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Journal Article Muscle Nerve · July 2019 INTRODUCTION: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, ... Full text Open Access Link to item Cite

A Phase 1 Randomized Study of Single Intravenous Infusions of the Novel Nitroxyl Donor BMS-986231 in Healthy Volunteers.

Journal Article J Clin Pharmacol · May 2019 Nitroxyl (HNO) is a reactive nitrogen molecule that has potential therapeutic benefits for patients with acute heart failure. The results of the first-in-human study for BMS-986231, a novel HNO donor, are reported. The aim of this sequential cohort study w ... Full text Link to item Cite

Treatment patterns and costs of chronic inflammatory demyelinating polyneuropathy: A claims database analysis

Journal Article American Health and Drug Benefits · May 1, 2019 BACKGROUND: Corticosteroids, plasma exchange, and intravenous immunoglobulin (IVIG) have been standard-of-care treatments for chronic inflammatory demyelinating polyneuropathy (CIDP) for more than 2 decades. Despite guideline recommendations for best clini ... Cite

Tacrolimus inhibits Th1 and Th17 responses in MuSK-antibody positive myasthenia gravis patients.

Journal Article Exp Neurol · February 2019 Muscle specific tyrosine kinase antibody positive myasthenia gravis (MuSK- MG) is characterized by autoantibodies against the MuSK protein of the neuromuscular junction resulting in weakness of bulbar and proximal muscles. We previously demonstrated that p ... Full text Link to item Cite

Two comparative assessments of intravenous immunoglobulin therapy switching patterns in the treatment of chronic inflammatory demyelinating polyneuropathy in the US

Journal Article Patient Preference and Adherence · January 1, 2019 Purpose: For chronic inflammatory demyelinating polyneuropathy (CIDP) patients, each branded intravenous immunoglobulin (IVIG) treatment differs in production processes, virus elimination, formulation, and composition. Given the limited availability of rea ... Full text Cite

Establishment of normative ranges of the healthy human immune system with comprehensive polychromatic flow cytometry profiling.

Journal Article PLoS One · 2019 Existing normative flow cytometry data have several limitations including small sample sizes, incompletely described study populations, variable flow cytometry methodology, and limited depth for defining lymphocyte subpopulations. To overcome these issues, ... Full text Open Access Link to item Cite

Antagonism of the Neonatal Fc Receptor as an Emerging Treatment for Myasthenia Gravis.

Journal Article Front Immunol · 2019 Myasthenia gravis is an autoimmune disease in which immunoglobulin G (IgG) autoantibodies are formed against the nicotinic acetylcholine receptor (AChR) or other components of the neuromuscular junction. Though effective treatments are currently available, ... Full text Link to item Cite

Circulating microRNA plasma profile in MuSK+ myasthenia gravis.

Journal Article J Neuroimmunol · December 15, 2018 Muscle-specific tyrosine kinase antibody positive myasthenia gravis (MuSK+ MG) is an immunological subtype with distinctive pathogenic mechanisms and clinical features. The aim of this study was to analyze the circulating plasma microRNA profile of patient ... Full text Link to item Cite

Population Pharmacokinetics and Exploratory Exposure-Response Relationships of Diazepam in Children Treated for Status Epilepticus.

Journal Article CPT Pharmacometrics Syst Pharmacol · November 2018 Diazepam is labeled for status epilepticus (SE) in children, but there are limited data characterizing its disposition in pediatric patients. We developed a population pharmacokinetic (PK) model of i.v. diazepam in children with SE. We evaluated relationsh ... Full text Link to item Cite

A Population-Based Pharmacokinetic Model Approach to Pantoprazole Dosing for Obese Children and Adolescents.

Journal Article Paediatr Drugs · October 2018 BACKGROUND AND AIMS: Pharmacokinetic data for proton pump inhibitors (PPIs), acid-suppression drugs commonly prescribed to children, are lacking for obese children who are at greatest risk for acid-related disease. In a recent multi-center investigation, w ... Full text Link to item Cite

Advances in autoimmune myasthenia gravis management.

Journal Article Expert Rev Neurother · July 2018 Myasthenia gravis (MG) is an autoimmune neuromuscular disorder with no cure and conventional treatments limited by significant adverse effects and variable benefit. In the last decade, therapeutic development has expanded based on improved understanding of ... Full text Link to item Cite

Validation of a simple disease-specific, quality-of-life measure for diabetic polyneuropathy: CAPPRI.

Journal Article Neurology · June 5, 2018 OBJECTIVE: We studied the performance of a 15-item, health-related quality-of-life polyneuropathy scale in the clinic setting in patients with diabetic distal sensorimotor polyneuropathy (DSPN). METHODS: Patients with DSPN from 11 academic sites completed ... Full text Link to item Cite

B cells in the pathophysiology of myasthenia gravis.

Journal Article Muscle Nerve · February 2018 Myasthenia gravis (MG) is an archetypal autoimmune disease. The pathology is characterized by autoantibodies to the acetylcholine receptor (AChR) in most patients or to muscle-specific tyrosine kinase (MuSK) in others and to a growing number of other posts ... Full text Link to item Cite

Comparative effectiveness clinical trials to advance treatment of myasthenia gravis.

Journal Article Ann N Y Acad Sci · February 2018 Myasthenia gravis (MG) presents many challenges for establishing treatment efficacy through clinical trials. Among these are the rarity and heterogeneity of the disease, spontaneous fluctuations, prolonged latency to effect for many immunosuppressive drugs ... Full text Link to item Cite

Obese Children Require Lower Doses of Pantoprazole Than Nonobese Peers to Achieve Equal Systemic Drug Exposures.

Journal Article J Pediatr · February 2018 OBJECTIVE: To assess appropriate pantoprazole dosing for obese children, we conducted a prospective pharmacokinetics (PK) investigation of pantoprazole in obese children, a patient population that is traditionally excluded from clinical trials. STUDY DESIG ... Full text Link to item Cite

Reliability of the triple-timed up-and-go test.

Journal Article Muscle Nerve · January 2018 INTRODUCTION: We report the reliability of a new measure, the triple-timed up-and-go (3TUG) test, for assessing clinical function in patients with Lambert-Eaton myasthenia (LEM). METHODS: Intrarater reproducibility and interrater agreement of the 3TUG test ... Full text Link to item Cite

Emerging therapeutics for myasthenia gravis

Conference Current Clinical Neurology · January 1, 2018 At no time in the history of myasthenia gravis has there been a time of greater therapeutic development. This situation likely stems from the well-defined pathophysiology of MG, which has made target identification relatively straightforward for drug devel ... Full text Cite

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.

Journal Article Lancet Neurol · December 2017 BACKGROUND: Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinic ... Full text Link to item Cite

Population Pharmacokinetics/Pharmacodynamics of 3,4-Diaminopyridine Free Base in Patients With Lambert-Eaton Myasthenia.

Conference CPT Pharmacometrics Syst Pharmacol · September 2017 Lambert-Eaton myasthenia (LEM) is a rare autoimmune disorder associated with debilitating muscle weakness. There are limited treatment options and 3,4-diaminopyridine (3,4-DAP) free base is an investigational orphan drug used to treat LEM-related weakness. ... Full text Link to item Cite

Population Pharmacokinetics and Exploratory Pharmacodynamics of Lorazepam in Pediatric Status Epilepticus.

Journal Article Clin Pharmacokinet · August 2017 BACKGROUND: Lorazepam is one of the preferred agents used for intravenous treatment of status epilepticus (SE). We combined data from two pediatric clinical trials to characterize the population pharmacokinetics of intravenous lorazepam in infants and chil ... Full text Link to item Cite

QMG and MG-ADL correlations: Study of eculizumab treatment of myasthenia gravis.

Journal Article Muscle Nerve · August 2017 INTRODUCTION: A phase 2 study of eculizumab for treating myasthenia gravis (MG) used the quantitative myasthenia gravis score (QMG) and myasthenia gravis activities of daily living profile (MG-ADL) to evaluate baseline disease severity and treatment respon ... Full text Link to item Cite

Phase 1 Randomized, Double-Blind, Placebo-Controlled Study to Determine the Safety, Tolerability, and Pharmacokinetics of a Single Escalating Dose and Repeated Doses of CN-105 in Healthy Adult Subjects.

Journal Article J Clin Pharmacol · June 2017 Spontaneous intracranial hemorrhage (ICH) remains a devastating stroke subtype, affecting as many as 80,000 people annually in the United States and associated with extremely high mortality. In the absence of any pharmacological interventions demonstrated ... Full text Link to item Cite

A Systematic Literature Review Approach to Estimate the Therapeutic Index of Selected Immunosuppressant Drugs After Renal Transplantation.

Journal Article Ther Drug Monit · February 2017 BACKGROUND: Drugs that exhibit close margins between therapeutic and toxic blood concentrations are considered to have a narrow therapeutic index (NTI). The Food and Drug Administration has proposed that NTI drugs should have more stringent bioequivalence ... Full text Link to item Cite

LEAN BODY WEIGHT (LBW) DOSING OF PANTOPRAZOLE IS APPROPRIATE FOR OBESE CHILDREN.

Conference CLINICAL PHARMACOLOGY & THERAPEUTICS · February 1, 2017 Link to item Cite

B10 Cell Frequencies and Suppressive Capacity in Myasthenia Gravis Are Associated with Disease Severity.

Journal Article Front Neurol · 2017 Myasthenia gravis (MG) is a T cell-dependent, B cell-mediated disease. The mechanisms for loss of self-tolerance in this disease are not well understood, and recently described regulatory B cell (Breg) subsets have not been thoroughly investigated. B10 cel ... Full text Open Access Link to item Cite

International clinimetric evaluation of the MG-QOL15, resulting in slight revision and subsequent validation of the MG-QOL15r.

Journal Article Muscle Nerve · December 2016 INTRODUCTION: The MG-QOL15 is a validated, health-related quality of life (HRQOL) measure for myasthenia gravis (MG). Widespread use of the scale gave us the opportunity to further analyze its clinimetric properties. METHODS: We first performed Rasch analy ... Full text Link to item Cite

Use of Therapeutic Drug Monitoring, Electronic Health Record Data, and Pharmacokinetic Modeling to Determine the Therapeutic Index of Phenytoin and Lamotrigine.

Journal Article Ther Drug Monit · December 2016 BACKGROUND: Defining a drug's therapeutic index (TI) is important for patient safety and regulating the development of generic drugs. For many drugs, the TI is unknown. A systematic approach was developed to characterize the TI of a drug using therapeutic ... Full text Link to item Cite

Effect of therapeutic plasma exchange on immunoglobulins in myasthenia gravis.

Journal Article Autoimmunity · November 2016 An integrated understanding of therapeutic plasma exchange (TPE) effects on immunoglobulins, autoantibodies, and natural or acquired (vaccine) protective antibodies in patients with autoimmune myasthenia gravis (MG) is lacking. Prior studies measured TPE e ... Full text Link to item Cite

Therapeutic Drug Monitoring, Electronic Health Records, and Pharmacokinetic Modeling to Evaluate Sirolimus Drug Exposure-Response Relationships in Renal Transplant Patients.

Journal Article Ther Drug Monit · October 2016 BACKGROUND: Sirolimus, an immunosuppressive agent used in renal transplantation, can prevent allograft rejection. Identification of the therapeutic index (the ratio of minimum toxic concentration to minimum therapeutic concentration) for immunosuppresants ... Full text Link to item Cite

A COMPARATIVE ASSESSMENT OF IVIG THERAPY IN THE TREATMENT OF CIDP

Conference JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM · September 1, 2016 Link to item Cite

SWITCHING PATTERNS IN PATIENTS WITH ICD-9 DIAGNOSED CIDP INITIATING IVIG TREATMENT

Conference JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM · September 1, 2016 Link to item Cite

AN EXTENDED VIEW OF TREATMENT PATTERNS IN CIDP

Conference JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM · September 1, 2016 Link to item Cite

Construction and validation of the chronic acquired polyneuropathy patient-reported index (CAP-PRI): A disease-specific, health-related quality-of-life instrument.

Journal Article Muscle Nerve · June 2016 INTRODUCTION: Generic health-related quality-of-life (HRQOL) patient-reported outcome measures have been used in patients with chronic immune-mediated polyneuropathies. We have created a disease-specific HRQOL instrument. METHODS: The chronic acquired poly ... Full text Link to item Cite

Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME): A randomized, controlled trial.

Journal Article Muscle Nerve · March 2016 INTRODUCTION: In this study we evaluated the safety, tolerability, and efficacy of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. METHODS: This investigation was a randomized, double-blind, placebo-cont ... Full text Link to item Cite

Adaptive immune response to therapy in hmgcr autoantibody myopathy.

Journal Article Muscle Nerve · February 2016 INTRODUCTION: We evaluated the response to immunosuppression in a case of 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR)-autoantibody myopathy. METHODS: T- and B-cell subsets were determined by flow cytometry pre- and posttherapy. RESULTS: Baselin ... Full text Link to item Cite

Current Treatment, Emerging Translational Therapies, and New Therapeutic Targets for Autoimmune Myasthenia Gravis.

Journal Article Neurotherapeutics · January 2016 Myasthenia gravis (MG) is an autoimmune disease associated with the production of autoantibodies against 1) the skeletal muscle acetylcholine receptor; 2) muscle-specific kinase, a receptor tyrosine kinase critical for the maintenance of neuromuscular syna ... Full text Link to item Cite

Therapeutic Index Estimation of Antiepileptic Drugs: A Systematic Literature Review Approach.

Journal Article Clin Neuropharmacol · 2016 OBJECTIVES: Our study aimed to determine whether data obtained from the medical literature can be used to estimate the therapeutic index of 5 antiepileptic drugs (AEDs): carbamazepine, lamotrigine, phenobarbital, phenytoin, and valproate. METHODS: We perfo ... Full text Link to item Cite

Characterization of B cells in muscle-specific kinase antibody myasthenia gravis.

Journal Article Neurol Neuroimmunol Neuroinflamm · April 2015 OBJECTIVE: To characterize B-cell subsets in patients with muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG). METHODS: In accordance with Human Immunology Project Consortium guidelines, we performed polychromatic flow cytometry and ELISA assays ... Full text Open Access Link to item Cite

Assessment of the effects of lacosamide on sleep parameters in healthy subjects.

Journal Article Seizure · February 2015 PURPOSE: Seizures and antiepileptic drugs (AED) may disrupt sleep patterns in patients with epilepsy, thus evaluation of lacosamide effects on objective and subjective sleep measures is warranted. METHODS: A multicenter, interventional, open-label study (N ... Full text Link to item Cite

Myasthenia gravis and Lambert-Eaton myasthenic syndrome.

Journal Article Continuum (Minneap Minn) · October 2014 PURPOSE OF REVIEW: This article reviews the clinical presentations, diagnostic findings, and treatment options for autoimmune myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome. RECENT FINDINGS: Immunologic research is unraveling the immunopathol ... Full text Link to item Cite

Characterization of CD4 and CD8 T cell responses in MuSK myasthenia gravis.

Journal Article J Autoimmun · August 2014 Muscle specific tyrosine kinase myasthenia gravis (MuSK MG) is a form of autoimmune MG that predominantly affects women and has unique clinical features, including prominent bulbar weakness, muscle atrophy, and excellent response to therapeutic plasma exch ... Full text Open Access Link to item Cite

Patient demographics and health plan paid costs in chronic inflammatory demyelinating polyneuropathy.

Conference Muscle Nerve · July 2014 INTRODUCTION: We determined health plan paid costs and healthcare resource usage of patients with chronic inflammatory demyelinating polyneuropathy (CIDP). METHODS: CIDP patients from 9 U.S. commercial health plans with claims in 2011 were identified from ... Full text Link to item Cite

Is the decremental pattern in Lambert-Eaton syndrome different from that in myasthenia gravis?

Journal Article Clin Neurophysiol · June 2014 OBJECTIVE: We reviewed our experience to determine if the decremental pattern during low frequency repetitive nerve stimulation (LF-RNS) distinguishes between the Lambert-Eaton myasthenic syndrome (LEMS) and myasthenia gravis (MG). METHODS: LF-RNS studies ... Full text Link to item Cite

Pharmacokinetics and pharmacodynamics of antifungals in children: clinical implications.

Journal Article Drugs · June 2014 Invasive fungal disease (IFD) remains life threatening in premature infants and immunocompromised children despite the recent development of new antifungal agents. Optimal dosing of antifungals is one of the few factors clinicians can control to improve ou ... Full text Link to item Cite

Stroke after primary percutaneous coronary intervention in patients with ST-segment elevation myocardial infarction: timing, characteristics, and clinical outcomes.

Journal Article Circ Cardiovasc Interv · April 2013 BACKGROUND: Stroke is a rare but potentially devastating complication of acute myocardial infarction. Little is known about stroke timing, characteristics, and clinical outcomes in patients with ST-segment elevation myocardial infarction treated with prima ... Full text Link to item Cite

A retrospective study of complications of therapeutic plasma exchange in myasthenia.

Journal Article Muscle Nerve · February 2013 INTRODUCTION: Venous access for therapeutic plasma exchange (TPE) in myasthenia gravis (MG) can be achieved by central venous catheters (CVC) or peripheral veins (PV), and the preferred method varies among providers. We evaluated our institutional experien ... Full text Link to item Cite

Recommendations for myasthenia gravis clinical trials.

Journal Article Muscle Nerve · June 2012 The recommendations for clinical research standards published in 2000 by a task force of the Medical Scientific Advisory Board (MSAB) of the Myasthenia Gravis Foundation of America (MGFA) were largely successful in introducing greater uniformity in the rec ... Full text Link to item Cite

Estimated cost of treating myasthenia gravis in an insured U.S. population.

Journal Article Muscle Nerve · March 2012 INTRODUCTION: In this study we estimated the costs paid by U.S. health plans for treating myasthenia gravis (MG) in 2009 and determined the major cost drivers. METHODS: One hundred thirteen MG patients were matched by propensity scores with 339 non-MG pati ... Full text Link to item Cite

Cost analysis of myasthenia gravis from a large U.S. insurance database.

Journal Article Muscle Nerve · December 2011 INTRODUCTION: Little is known about the costs of managing rare diseases, and comprehensive healthcare costs have not been reported for myasthenia gravis (MG). We evaluated the direct costs and healthcare resource utilization in insured MG patients. METHODS ... Full text Link to item Cite

Anti-MuSK antibody myasthenia gravis: clinical findings and response to treatment in two large cohorts.

Journal Article Muscle Nerve · July 2011 INTRODUCTION: Myasthenia gravis (MG) patients with autoantibodies to muscle-specific tyrosine kinase (MuSK) represent a distinct subset of those with this disease. Treatment and outcomes data in these patients are limited and conflicting. METHODS: We revie ... Full text Link to item Cite

Update on muscle-specific tyrosine kinase antibody positive myasthenia gravis.

Journal Article Curr Opin Neurol · October 2010 PURPOSE OF REVIEW: Important concepts regarding the pathogenesis, clinical features, diagnosis and treatment of muscle-specific tyrosine kinase (MuSK) antibody positive myasthenia gravis will be reviewed. Special attention will be paid to clinical phenotyp ... Full text Link to item Cite

Use of levetiracetam in subarachnoid hemorrhage

Conference EPILEPSIA · October 1, 2007 Link to item Cite

[3H]-flunitrazepam-labeled benzodiazepine binding sites in the hippocampal formation in autism: a multiple concentration autoradiographic study.

Journal Article J Autism Dev Disord · May 2007 Increasing evidence indicates that the GABAergic system in cerebellar and limbic structures is affected in autism. We extended our previous study that found reduced [(3)H]flunitrazepam-labeled benzodiazepine sites in the autistic hippocampus to determine w ... Full text Link to item Cite

Treatment outcome and outcome associations in children with pervasive developmental disorders treated with selective serotonin reuptake inhibitors: a chart review.

Journal Article J Child Adolesc Psychopharmacol · 2006 PURPOSE: The aim of this study was to determine the outcome and predictors of outcome with selective serotonin reuptake inhibitors (SSRIs) in outpatient children and adolescents with pervasive developmental disorders (PDDs). METHOD: Clinic charts were revi ... Full text Link to item Cite

Density and distribution of hippocampal neurotransmitter receptors in autism: an autoradiographic study.

Journal Article J Autism Dev Disord · December 2001 Neuropathological studies in autistic brains have shown small neuronal size and increased cell packing density in a variety of limbic system structures including the hippocampus, a change consistent with curtailment of normal development. Based on these ob ... Full text Link to item Cite