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For information on Neuromuscular clinical trials, please contact Kristiana Salmon.


Current Recruiting Studies


ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
Baxalta 161403
A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human), 10% (GAMMAGARD LIQUID/KIOVIG) for the Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

A Phase 3 Study of Amifampridine Phosphate in Patients with Congenital Myasthenic Syndromes (CMS)

A Phase 2, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects with Generalized Myasthenia Gravis

Investigating Pompe Prevalence in NEuromuscular Medicine Academic Practices (IPANEMA Study)
Genzyme Pompe (disease registry)
The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.


Sanofi EFC14028 Late Onset Pompe
A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease