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Neuromuscular Medicine

Clinical Trials

Below is a list of UCLA Neurology’s ongoing trials in neuromuscular diseases
Click the link to for the most up-to-date information

Duchenne Muscular Dystrophy

For additional information, please visit the Center for Duchenne Muscular Dystrophy at UCLA website.

Spinal Muscular Atrophy

Phase 3 Study of nusinersen in Infantile-onset Spinal Muscular Atrophy - Sponsored by IONIS/Biogen, Inc. – extension study ongoing
Phase 3 study of nusinersen in type 2 Spinal Muscular Atrophy, Sponsored by IONIS/Biogen Pharmaceuticals, Inc. – exten sion study ongoing
Phase 2 study of nusinersen in patients with spinal muscular atrophy who are not eligible to participate in other nusinersen studies, Sponsored by Biogen/IONIS – extension study ongoing
Phase 2 study of CK-2127107 (a troponin activator) In Spinal Muscular Atrophy, Sponsored by Cytokinetics – study ongoing
Phase 1, Intrathecal Gene Therapy (AAV9-SMN) study for Type 2 Spinal Muscular Atrophy, Sponsored by Avexis – soon to start
Phase 3, Intravenous Gene Therapy (AAV9-SMN) study for Type 1 Spinal Muscular Atrophy, Sponsored by Avexis – soon to start

GNE myopathy

Phase 2 Study of Sialic Acid-Extended Release in Patients with GNE Myopathy or Hereditary Inclusion Body Myopathy” Sponsored by Ultragenyx Pharmaceutical – extension study ongoing

Mitochondrial Myopathy

Phase 2 study of RTA 408 In Mitochondrial Myopathy Sponsored by Reata Pharmaceuticals, Inc. – study ongoing


Expanded Access Program for 3,4-Diaminopyridine Phosphate in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndrome Sponsored by Catalyst Pharmaceutical Partners, Inc. – study ongoing
Phase 3 Follow-Up Study of 3,4 Diaminopyridine Phosphate in Lambert-Eaton Myasthenic Syndrome, Sponsored by Catalyst Pharmaceuticals, Inc. – study ongoing
Phase 3 study of 3,4-Diaminopyridine Phosphate Congenital Myasthenic Syndromes Sponsored by Catalyst – study ongoing

Myotubular Myopathy

Pre-phase 1, Natural History study of X-Linked Myotubular Myopathy, Sponsored by Audentes Therapeutics - study ongoing
Phase 1/2, Gene Therapy (AAV8-MTM) in X-Linked Myotubular Myopathy (XLMTM) Sponsored by Audentes Therapeutics – soon to start


Phase 2 study of ACE-083 (anti-myostatin) in Facioscapulohumeral Muscular Dystrophy Sponsored by Acceleron – soon to start

Pompe Disease

Phase 3 of neoGAA (GZ402666) vs. lumizyme enzyme replacement in treatment-naïve patients with late-onset Pompe disease Sponsored by Sanofi – soon to start


UCLA is part of the NeuroNEXT network that is sponsored by the National Institute of Neurological Diseases and Stroke at the National Institute of Health. Dr. Perry Shieh and Michael Graves participate in a number of clinical trials that are sponsored by NIH, industry, and non-profit organizations. Currently active clinical trials include trials for: