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 ...

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 ...

A recent discovery, the highly adaptable CRISPR–Cas9 system, is already finding widespread use for cell therapy and in vivo gene editing Ongoing challenges include efficient gene delivery, ...

Muscle ultrasound has emerged as a pivotal, non‐invasive imaging modality that offers real‐time insights into muscle structure and pathology. This technique enables both qualitative and quantitative ...

There are more than 200 known neuromuscular diseases affecting over 400,000 people in the United States alone. Among these are "Lou Gehrig's Disease" (amyotrophic lateral sclerosis, or ALS), the ...