UPMC Medical Education (Pittsburgh) Neuromuscular Medicine (Neurology)

Topics

• Muscle Physiology and Disorders 91
• Amyotrophic Lateral Sclerosis Research 32
• Inflammatory Myopathies and Dermatomyositis 28
• Myasthenia Gravis and Thymoma 20
• Virus-based gene therapy research 16
• Peripheral Neuropathies and Disorders 12
• Genetic Neurodegenerative Diseases 9
• Lysosomal Storage Disorders Research 9
• Amyloidosis: Diagnosis, Treatment, Outcomes 7
• Neurogenetic and Muscular Disorders Research 6
• Cardiomyopathy and Myosin Studies 5
• Nerve injury and regeneration 5

Most cited

1. Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.

   Neurology · 2024 · 70 citations · Paula Clemens

2. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.

   The Lancet. Neurology · 2021 · 153 citations · Paula Clemens

3. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

   JAMA neurology · 2022 · 114 citations · Paula Clemens

4. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

   JAMA network open · 2022 · 76 citations · Paula Clemens

5. First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy.

   Nature medicine · 2025 · 22 citations · Paula Clemens

6. ALS-associated KIF5A mutations abolish autoinhibition resulting in a toxic gain of function.

   Cell reports · 2022 · 88 citations · Jonathan Brent