Joseph Patrick Horrigan

Journal Article Pharmaceutics · August 1, 2025 Background: GSK3β is an intracellular regulatory kinase that is dysregulated in multiple tissues in Type 1 myotonic dystrophy (DM-1). Tideglusib inhibits GSK3β activity in preclinical models of DM-1 and promotes cellular maturation, normalising aberrant mo ... Full text Cite

An open-label study evaluating the safety and efficacy of AMO-01 for the treatment of seizures in Phelan-McDermid syndrome.

Journal Article HGG Adv · April 10, 2025 Phelan-McDermid syndrome (PMS) is a neurodevelopmental disorder caused by haploinsufficiency of the SHANK3 gene. Approximately 25% of individuals with PMS have epilepsy. Treatment of epilepsy in PMS may require multiple anticonvulsants, and in a minority o ... Full text Link to item Cite

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Journal Article Trials · December 21, 2024 INTRODUCTION: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of cli ... Full text Link to item Cite

Ensuring Stakeholder Feedback in the Design and Conduct of Clinical Trials for Rare Diseases: ISCTM Position Paper of the Orphan Disease Working Group.

Journal Article Innov Clin Neurosci · 2024 The 1983 Orphan Drug Act in the United States (US) changed the landscape for development of therapeutics for rare or orphan diseases, which collectively affect approximately 300 million people worldwide, half of whom are children. The act has undoubtedly a ... Link to item Cite

Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond: Second of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development.

Journal Article Innov Clin Neurosci · 2023 This article expands upon a session, titled "Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond," that was presented as part of a two-day meeting on pediatric drug development at the International Society for Central ... Link to item Cite

Development of trofinetide for the treatment of Rett syndrome: from bench to bedside.

Journal Article Front Pharmacol · 2023 Rett syndrome (RTT) is rare neurodevelopmental disorder caused by mutations in the MECP2 gene that encodes methyl-CpG-binding protein 2 (MeCP2), a DNA-binding protein with roles in epigenetic regulation of gene expression. Functional loss of MeCP2 results ... Full text Link to item Cite

Respiratory Effects of the Atypical Tricyclic Antidepressant Tianeptine in Human Models of Opioid-induced Respiratory Depression.

Journal Article Anesthesiology · October 1, 2022 BACKGROUND: Animal data suggest that the antidepressant and α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor modulator tianeptine is able to prevent opioid-induced respiratory depression. The hypothesis was that oral or intravenous tianeptine ... Full text Link to item Cite

Clinician-and Patient-reported Endpoints in CNS Orphan Drug Clinical Trials: ISCTM Position Paper on Best Practices for Endpoint Selection, Validation, Training, and Standardization

Journal Article Innovations in Clinical Neuroscience · October 1, 2021 Objective: The International Society of CNS Clinical Trials Methodology (ISCTM) Working Group on Rare Disease/Orphan Drug Development is dedicated to improving and streamlining trials to best develop new treatments for rare diseases. The rarity of these di ... Cite

A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1).

Journal Article Pediatr Neurol · November 2020 BACKGROUND: GSK3β is an intracellular regulatory kinase that is dysregulated in multiple tissues in type 1 myotonic dystrophy, a rare neuromuscular disorder that manifests at any age. AMO-02 (tideglusib) inhibits GSK3β activity in preclinical models of typ ... Full text Link to item Cite

A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome.

Journal Article Pediatr Neurol · September 2020 BACKGROUND: We analyze the safety and tolerability of trofinetide and provide a preliminary evaluation of its efficacy in adolescent and adult males with fragile X syndrome. METHODS: This study was an exploratory, phase 2, multicenter, double-blind, placeb ... Full text Link to item Cite

A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder.

Journal Article J Pediatr · July 2020 OBJECTIVE: To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD). STUDY DESIGN: This prospective, randomized, placebo-controlled, doubl ... Full text Open Access Link to item Cite

Population pharmacokinetics of NNZ-2566 in healthy subjects.

Journal Article Eur J Pharm Sci · November 15, 2017 NNZ-2566 is a novel, small molecule being developed as a treatment for cognitive impairment in different CNS conditions, including Rett and Fragile-X syndrome, both of which are associated with moderate to severe neurodevelopmental disorder. In the current ... Full text Link to item Cite

A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome.

Journal Article Pediatr Neurol · November 2017 BACKGROUND: This study aimed to determine the safety and tolerability of trofinetide and to evaluate efficacy measures in adolescent and adult females with Rett syndrome, a serious and debilitating neurodevelopmental condition for which no therapies are av ... Full text Link to item Cite

5.16 AMO-01 in the SHANK3 Knockout Transgenic Mouse Model of Phelan Mcdermid Syndrome

Conference Journal of the American Academy of Child & Adolescent Psychiatry · October 2017 Full text Cite

Assessment of Caregiver Inventory for Rett Syndrome.

Journal Article J Autism Dev Disord · April 2017 Rett syndrome (RTT) requires total caregiver attention and leads to potential difficulties throughout life. The Caregiver Burden Inventory, designed for Alzheimer disease, was modified to a RTT Caregiver Inventory Assessment (RTT CIA). Reliability and face ... Full text Link to item Cite

Adjunctive Maintenance Lamotrigine for Pediatric Bipolar I Disorder: A Placebo-Controlled, Randomized Withdrawal Study.

Journal Article J Am Acad Child Adolesc Psychiatry · December 2015 OBJECTIVE: This study aimed to compare the efficacy of lamotrigine versus placebo in 10- to 17-year-olds with bipolar I disorder (BP-I) who were receiving conventional bipolar disorder treatment. METHOD: In this randomized withdrawal trial, patients with B ... Full text Link to item Cite

Improving Treatment Trial Outcomes for Rett Syndrome: The Development of Rett-specific Anchors for the Clinical Global Impression Scale.

Journal Article J Child Neurol · November 2015 Rett syndrome is a genetically based neurodevelopmental disorder. Although the clinical consequences of Rett syndrome are profound and lifelong, currently no approved drug treatments are available specifically targeted to Rett symptoms. High quality outcom ... Full text Link to item Cite

Measuring social communication behaviors as a treatment endpoint in individuals with autism spectrum disorder.

Journal Article Autism · July 2015 Social communication impairments are a core deficit in autism spectrum disorder. Social communication deficit is also an early indicator of autism spectrum disorder and a factor in long-term outcomes. Thus, this symptom domain represents a critical treatme ... Full text Link to item Cite

A Phase II study of a histamine H₃ receptor antagonist GSK239512 for cognitive impairment in stable schizophrenia subjects on antipsychotic therapy.

Journal Article Schizophr Res · May 2015 This Phase II exploratory study assessed GSK239512, a brain penetrant histamine H₃ receptor antagonist, versus placebo on cognitive impairment in 50 stable outpatients with schizophrenia. Subjects were randomized to placebo or GSK239512 for 7 weeks (4 week ... Full text Link to item Cite

Measuring repetitive behaviors as a treatment endpoint in youth with autism spectrum disorder.

Journal Article Autism · January 2015 Restricted interests and repetitive behaviors vary widely in type, frequency, and intensity among children and adolescents with autism spectrum disorder. They can be stigmatizing and interfere with more constructive activities. Accordingly, restricted inte ... Full text Link to item Cite

Measuring anxiety as a treatment endpoint in youth with autism spectrum disorder.

Journal Article J Autism Dev Disord · May 2014 Despite the high rate of anxiety in individuals with autism spectrum disorder (ASD), measuring anxiety in ASD is fraught with uncertainty. This is due, in part, to incomplete consensus on the manifestations of anxiety in this population. Autism Speaks asse ... Full text Link to item Cite

A randomized, double-blind, placebo-controlled, 16-week study of the H3 receptor antagonist, GSK239512 as a monotherapy in subjects with mild-to-moderate Alzheimer's disease.

Journal Article Curr Alzheimer Res · January 2014 INTRODUCTION: Histaminergic H3 receptors may play a role in modulating cholinergic and monoaminergic neurotransmission. This Phase II study evaluated the efficacy and safety of GSK239512, a highly potent, brain penetrant H3 receptor antagonist as monothera ... Full text Link to item Cite

Leukocyte counts in cerebrospinal fluid and blood following firategrast treatment in subjects with relapsing forms of multiple sclerosis.

Journal Article Eur J Neurol · July 2013 BACKGROUND AND PURPOSE: Firategrast is an orally bioavailable alpha4 beta1/alpha4 beta7 integrin antagonist designed to reduce trafficking of lymphocytes into the central nervous system (CNS). This could decrease multiple sclerosis (MS) activity, but may c ... Full text Link to item Cite

Measuring Anxiety as a Treatment Endpoint in Youth with Autism Spectrum Disorder

Journal Article Journal of Autism and Developmental Disorders · 2013 Cite

Randomized, Double-Blind, Placebo Controlled, Parallel Group Exploratory Study of GSK239512 for Cognitive Impairment in Stable Schizophrenia Subjects on Background Antipsychotic Therapy

Conference BIOLOGICAL PSYCHIATRY · April 15, 2012 Link to item Cite

Firategrast for relapsing remitting multiple sclerosis: a phase 2, randomised, double-blind, placebo-controlled trial.

Journal Article Lancet Neurol · February 2012 BACKGROUND: Monoclonal antibody therapy against α4β-integrin is efficacious in patients with multiple sclerosis (MS) with some safety concerns. We assessed the safety and efficacy of firategrast, a small oral anti-α4β-integrin molecule, in patients with re ... Full text Link to item Cite

Clinical features of patients with treatment-emergent suicidal behavior following initiation of paroxetine therapy.

Journal Article J Affect Disord · January 2010 BACKGROUND: Understanding suicidal behavior is an important component of assessing suicidality in psychiatric patients. GlaxoSmithKline (GSK) conducted a meta-analysis of randomized, placebo-controlled trials to compare suicidality in adult patients treate ... Full text Link to item Cite

From trial and error to trial simulation. Part 2: an appraisal of current beliefs in the design and analysis of clinical trials for antidepressant drugs.

Journal Article Clin Pharmacol Ther · September 2009 Study design factors are partly to blame for the high failure rate in trials with antidepressant drugs. Clinical trial simulation (CTS) allows the investigation of the influence of design characteristics on important aspects of clinical trials such as powe ... Full text Link to item Cite

Gepirone extended-release in the treatment of adult outpatients with major depressive disorder: a double-blind, randomized, placebo-controlled, parallel-group study.

Journal Article J Clin Psychiatry · April 2008 OBJECTIVE: To evaluate the efficacy and tolerability of extended-release gepirone (gepirone-ER), a 5-HT(1A) agonist, versus placebo in the treatment of adult outpatients with major depressive disorder (MDD). METHOD: A double-blind, randomized, placebo-cont ... Full text Link to item Cite

A pilot study of an electronic, adolescent version of the quick inventory of depressive symptomatology.

Journal Article J Clin Psychiatry · September 2007 BACKGROUND: Adolescent depression assessments are time-intensive, often requiring separate interviews with an adolescent and a parent/ informant. In adults, a self-rated, interactive voice response (IVR) version of the Quick Inventory of Depressive Symptom ... Full text Link to item Cite

Analysis of electrocardiographic data following use of paroxetine in pediatric depression and obsessive-compulsive disorder.

Journal Article J Am Acad Child Adolesc Psychiatry · April 2006 OBJECTIVE: This retrospective analysis of electrocardiographic (ECG) data investigated the cardiovascular effects of paroxetine 10-50 mg/day in pediatric patients (7-18 years of age). Data were collected from three 8- to 10-week, randomized, placebo-contro ... Full text Link to item Cite

Bupropion extended release compared with escitalopram: Effects on sexual functioning and antidepressant efficacy in 2 randomized, double-blind, placebo-controlled studies

Journal Article Journal of Clinical Psychiatry · January 1, 2006 Objective: To compare the effects on sexual functioning and the antidepressant efficacy of once-daily bupropion extended release (XL) and escitalopram in adults with major depressive disorder (MDD). Method: Adult outpatients with moderate to severe DSM-IV- ... Full text Cite

Bupropion XL in adults with attention-deficit/hyperactivity disorder: a randomized, placebo-controlled study.

Journal Article Biol Psychiatry · April 1, 2005 BACKGROUND: Data remain limited on treatment strategies for adults with attention-deficit/hyperactivity disorder (ADHD). This study evaluated the efficacy and safety of an extended-release, once-daily formulation of bupropion (XL) in the treatment of adult ... Full text Link to item Cite

Oral flower essences for ADHD.

Journal Article J Am Acad Child Adolesc Psychiatry · August 2002 Full text Link to item Cite

Tourette's syndrome and autism: A search for common ground

Journal Article Mental Health Aspects of Developmental Disabilities · March 14, 2002 The comorbidity between autistic spectrum disorders and Tourette's syndrome (TS) raises intriguing questions about the neurobiology of both disorders. Repetitive movements, stereotypies, echophenomena, self-injurious behaviors and compulsive behaviors are ... Cite

Adderall, the atypicals, and weight gain.

Journal Article J Am Acad Child Adolesc Psychiatry · June 2001 Full text Link to item Cite

Present and future pharmacotherapeutic options for adult attention deficit/hyperactivity disorder.

Journal Article Expert Opin Pharmacother · April 2001 Attention deficit/hyperactivity disorder (ADHD) is often a lifelong condition. When untreated or undertreated, it appears to have a deleterious impact upon the daily functioning of the majority of adults that were diagnosed with this condition during child ... Full text Link to item Cite

Fluvoxamine and enuresis.

Journal Article J Am Acad Child Adolesc Psychiatry · December 2000 Full text Link to item Cite

Naltrexone treatment of self-injurious behavior.

Journal Article J Am Acad Child Adolesc Psychiatry · September 2000 Full text Link to item Cite

Low-dose amphetamine salts and adult attention-deficit/hyperactivity disorder.

Journal Article J Clin Psychiatry · June 2000 BACKGROUND: Effective treatments for attention-deficit/hyperactivity disorder (ADHD) in adults are still being defined. Pediatric studies have suggested that a mixed amphetamine salt product (Adderall) is safe and effective in the treatment of childhood fo ... Full text Link to item Cite

Naltrexone treatment of self-injurious behavior

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 2000 Full text Cite

Fluvoxamine and enuresis

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 2000 Full text Cite

Priapism associated with venlafaxine use.

Journal Article J Am Acad Child Adolesc Psychiatry · January 2000 Full text Link to item Cite

Nutritional supplements in ADHD.

Journal Article J Am Acad Child Adolesc Psychiatry · October 1999 Full text Link to item Cite

Guanfacine and secondary mania in children.

Journal Article J Affect Disord · August 1999 INTRODUCTION: Guanfacine hydrochloride is an alpha-2 adrenergic agonist, which has gained recent attention in the field of child and adolescent psychiatry. This medication has been described as effective in the management of attention-deficit hyperactivity ... Full text Link to item Cite

Appetite and weight in children treated for ADHD.

Journal Article J Am Acad Child Adolesc Psychiatry · July 1999 Full text Link to item Cite

Dystonia as a side effect of nonneuroleptics.

Journal Article J Am Acad Child Adolesc Psychiatry · July 1999 Full text Link to item Cite

Dystonia as a side effect of nonneuroleptics

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 1999 Full text Cite

Dr. Horrigttn replies

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 1999 Full text Cite

Comment by Dr. Horrigan, at the invitation of the editor

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 1999 Full text Cite

Seizure associated with bupropion and guanfacine.

Journal Article J Am Acad Child Adolesc Psychiatry · January 1999 Full text Link to item Cite

Diet and the atypical neuroleptics.

Journal Article J Am Acad Child Adolesc Psychiatry · November 1998 Full text Link to item Cite

Bupropion and guanfacine.

Journal Article J Am Acad Child Adolesc Psychiatry · July 1998 Full text Link to item Cite

Alternative therapies in the child psychiatric clinic.

Journal Article J Child Adolesc Psychopharmacol · 1998 Full text Link to item Cite

Does guanfacine trigger mania in children?

Journal Article J Child Adolesc Psychopharmacol · 1998 Full text Link to item Cite

Olanzapine in PDD.

Journal Article J Am Acad Child Adolesc Psychiatry · September 1997 Full text Link to item Cite

More on melatonin.

Journal Article J Am Acad Child Adolesc Psychiatry · August 1997 Full text Link to item Cite

More on melatonin.

Journal Article J Am Acad Child Adolesc Psychiatry · August 1997 Full text Link to item Cite

Risperidone and explosive aggressive autism.

Journal Article J Autism Dev Disord · June 1997 Many autistic patients with mental retardation have difficulties with explosivity and aggression. They often prove resistant to various pharmacotherapeutic interventions. In this study, 11 male outpatients (mean 18.3 years) were administered risperidone in ... Full text Link to item Cite

Letter to editor

Journal Article Journal of the American Academy of Child and Adolescent Psychiatry · January 1, 1997 Cite

The suppression of nightmares with guanfacine.

Journal Article J Clin Psychiatry · August 1996 Link to item Cite

Guanfacine for PTSD nightmares.

Journal Article J Am Acad Child Adolesc Psychiatry · August 1996 Full text Link to item Cite

Guanfacine for Treatment of Attention-Deficit Hyperactivity Disorder in Boys

Journal Article Journal of Child and Adolescent Psychopharmacology · January 1, 1995 The use of guanfacine hydrochloride (Tenex), a long-acting alpha2 agonist with a more favorable side effect profile relative to clonidine, was examined in boys with ADHD. In this prospective, open-label, non-blinded trial, guanfacine was prescri ... Full text Cite

Paroxetine-pimozide drug interaction.

Journal Article J Am Acad Child Adolesc Psychiatry · September 1994 Full text Link to item Cite