Neuromuscular diseases are conditions that affect your nerve and muscle function. Neuromuscular disorders affect your peripheral nerves—the nerves outside your brain and spinal cord. Types of ...
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 ...
Type 1 spinal muscular atrophy (SMA) is a progressive neuromuscular disease characterized by an onset at 6 months of age or younger, an inability to sit without support, and deficient levels of ...
Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...
A retrospective review of children with neuromuscular disorders and confirmed COVID-19 infection found the vast majority of patients experienced asymptomatic or mild disease, with no reported cases of ...
"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...
TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)--Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases ...
Girlband star Jesy Nelson revealed in an emotional Instagram video that both of her twins had been diagnosed with a genetic condition called Spinal Muscular Atrophy (SMA), type 1. U.K.-based Nelson, ...
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