ALS and Motor Neuron Disease
Ataxia
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Fascioscapulohumeral Muscular Dystrophy (FSHD)
Myotonic Dystrophy Type 1
Other Conditions
Refractory Generalized Myasthenia Gravis (GMG)
Sporadic Inclusion Body Myositis (IBM)
ALS and Motor Neuron Disease
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HEALEY ALS Platform Trial
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AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (CENTAUR)
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Skin Fibroblast Isolation for the Derivation of Induced-Pluripotent Stem Cells for Tissue Culture Models of Neurological Disease
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Study of Soluble Factors in the Cerebral Spinal Fluid in Individuals With Motor Neuron Disease
Ataxia
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RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe
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Clinical Outcome Measures in Friedreich’s Ataxia
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A Phase 2 Study of the Safety, Efficacy and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
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A Double-Blind, Randomized, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Fingolimod 0.5 Mg Administered Orally Once Daily Versus Placebo in Patients With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
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Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group, Phase III Study to Investigate the Efficacy, Safety and Tolerability of 2 Different Doses of Igpro20 (Subcutaneous Immunoglobulin) for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) – The PATH Study
Fascioscapulohumeral Muscular Dystrophy (FSHD)
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Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
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Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
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Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
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Preclinical Trial Preparedness in Fascioscapulohumeral Muscular Dystrophy (FSHD)
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A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies
Myotonic Dystrophy Type 1
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Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
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A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients With Myotonic Dystrophy Type 1
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A Multicenter Observational Study to Assess the Variability of Molecular Biomarkers and Clinical Measures in Patients With Myotonic Dystrophy Type 1
Other Conditions
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Transthyretin Amyloidosis Outcome Survey (THAOS)
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The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
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Topiramate for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN) (TopCSPN)
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Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)
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Lenalidomide in Anti-MAG Neuropathy: Phase 1b Study
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PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD
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HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
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A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
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Adults With SMA Treated With Nusinersen
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Adults With SMA Treated With Nusinersen
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Genetic and Molecular Characterization of Inherited Neuropathies
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A Double Blind, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, Efficacy and Preliminary Dose-Response of BAF312 in Patients With Active Dermatomyositis
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A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects With Late-Onset Pompe Disease
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Open-Label Trial of Ranolazine in Myotonia Congenita
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A Two-Part, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
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Transthyretin-Associated Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin (TTR) Mutations or Wild-Type TTR Amyloidosis
Refractory Generalized Myasthenia Gravis (GMG)
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Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
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Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
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A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
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Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)
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A Study of TAK-079 in People with Generalized Myasthenia Gravis
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Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE-XT)
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A Phase II Trial of Rituximab in Myasthenia Gravis
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A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (GMG)
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A Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacodynamics of Belimumab in Subjects With Generalized Myasthenia Gravis
Sporadic Inclusion Body Myositis (IBM)
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Study of Arimoclomol in Inclusion Body Myositis (IBM)
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A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel Group, Dose-Finding, Pivotal, Phase IIb/III Study to Evaluate the Efficacy, Safety and Tolerability of Intravenous BYM338 at 52 Weeks on Physical Function, Muscle Strength and Mobility and Additional Long-Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis
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Using Next Generation Sequencing to Unravel the Pathogenesis of Sporadic Inclusion Body Myositis (IBM) – The International IBM Consortium Genetic Study