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Undiagnosed conditions: Faculty in the UCLA Neuromuscular Medicine Program specialize in patients with undiagnosed conditions. These conditions may be genetic or acquired. Different diagnostic tools may need to be employed, including specialized electrodiagnostic testing, genetic testing, muscle and/or nerve biopsy, and muscle imaging (MRI, ultrasound, etc). If you or someone you know has an undiagnosed neuromuscular condition, you should ask your physician to refer to our clinic.
Services available through our clinic include:
Rare conditions: Rare neuromuscular conditions will sometimes require specialized treatment, including medical therapeutics, rehabilitative care, preventive care, and interdisciplinary medicine. The UCLA Neuromuscular program maintains updated treatment protocols that focus on state-of-the-art treatment for rare conditions.
To schedule an appointment, please call 310-825-7266.
The UCLA Neuropathy Clinic provides comprehensive evaluation, sophisticated tests, and state-of-the-art multidisciplinary care for patients with acquired and/or hereditary peripheral nerve disorders (motor, sensory, autonomic).
Acquired diseases:
Hereditary diseases:
To schedule an appointment, please call: 310-825-7266 or email the neuromuscular coordinator, Yvette Johnson, at YJohnson@mednet.ucla.edu.
These clinics provide state-of-the-art care for adult and pediatric patients who have been diagnosed with muscular dystrophy and related disorders. These may include:
Center For Duchenne Muscular Dystrophy at UCLA Pediatric Neuromuscular Clinic:
This clinic is sponsored by the Center for Duchenne Muscular Dystrophy at UCLA, the Muscular Dystrophy Association and Parent Project Muscular Dystrophy. This clinic is a Certified California Children's Services Care Center. This clinic is a multidisciplinary clinic with multiple specialties in attendance including:
This clinic meets three times month on designated Wednesday afternoons at Ronald Regan UCLA Medical Center. Patients in this clinic will be given the opportunity to participate in clinical trials for muscular dystrophy and other neuormuscular disorders as well as research that is coordinated through the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA. Patients that are interested in clinical trials may look to see the list of neuromuscular clinical trials offered by UCLA.
To schedule an appointment in this clinic, please call 310-825-7266 or email Jenna Gewirtz, the nurse coordinator.
Adult Muscular Dystrophy Clinic:
This clinic is sponsored by the Muscular Dystrophy Association.
The adult muscular clinic meets twice a month on designated Wednesday afternoons at Ronald Reagan UCLA Medical Center. Patients in this clinic will be given the opportunity to participate in clinical trials for Muscular Dystrophy. Patients that are interested in clinical trials may look to see the list of neuromuscular clinical trials offered by UCLA.
To schedule an appointment in this clinic, please call 310-825-7266 or email Jenna Gewirtz, the nurse coordinator.
ALS Clinic:
Clinic is for new patients only: Every second and fourth Thursday mornings.
Multi-specialty clinic for return patients every first, third and fifth Thursdays (full day).
Clinic contact: 310-794-1195
Clinic director: Martina Wiedau, MD
Emeritus neurologist: Michael Graves, MD
Clinic coordinators: Jacqui Parker and Yvette Johnson
Speech pathologist: Nancy Sedat
Occupational therapist: Sung Yu
Physical therapist: Sue Vereb
Respiratory therapist: Diana Guth
Social worker: Hillary Zebberman
Below is a partial list of UCLA Neurology’s ongoing trials in neuromuscular diseases
Click the link to ClinicalTrials.gov for the most up-to-date information
Duchenne Muscular Dystrophy
Clinical Trials.gov
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. – extension 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
LEMS
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
FSHD
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 Institutes 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:
