The American Journal of Managed Care

Shaping Gene Therapy Approaches in Neuromuscular Disease

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, and Advocacy

New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...
Business Wire

Neuromuscular Electrical Stimulation as a Treatment for Obstructive Sleep Apnea Gains Traction With a Recently Announced World Sleep 2023 Congress Symposium

BOSTON & LONDON--(BUSINESS WIRE)--Signifier Medical Technologies LLC (“Signifier” or the “Company”), a Boston- and London-based medical technology company, is excited to announce that the presentation ...
Carnegie Mellon University

Breakthrough in RNA Research Could Lead to Treatment for Neuromuscular Disorders

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...
The American Journal of Managed Care

Therapy Targeting Neuromuscular Junction Leads to SMA Improvement

New data suggest motor function improves with amifampridine. New phase 2 data for the spinal muscular atrophy (SMA) therapy amifampridine (Ruzurgi) suggests the drug is safe and effective at improving ...
The Anniston Star

Understanding Autoimmune Neuromuscular Diseases: Signs, Causes & Treatment

THURSDAY, Oct. 23, 2025 (HealthDay News) — Autoimmune neuromuscular diseases may sound complex, but understanding them is the first step to getting the right care and support. Conditions such as ...
Becker's Hospital Review

Treatment for rare degenerative neuromuscular disease earns orphan drug status

The Food and Drug Administration granted orphan drug status to Firdapse, an experimental treatment for patients with myasthenia gravis, a degenerative neuromuscular disease similar to Parkinson’s or ...

Structure-based RNA could lead to treatment for neuromuscular disorders

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...
MarketWatch

FDA Approves Amgen's UPLIZNA(R) (inebilizumab-cdon) for Generalized Myasthenia Gravis (gMG), Expanding Treatment Options for Patients Living with Autoimmune Neuromuscular Disease

The MarketWatch News Department was not involved in the creation of this content. New York, Dec. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) recognizes the U.S. Food and Drug ...
Business Wire

The Canadian Neuromuscular Disease Registry and Amylyx Pharmaceuticals Partner on Real-World Evidence Generation for a Novel Investigational Therapy in ALS with the Goal of ...

CALGARY, Alberta & TORONTO--(BUSINESS WIRE)--The Canadian Neuromuscular Disease Registry (CNDR), an independent, academic registry based out of the University of Calgary, and Amylyx Pharmaceuticals ...