For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne ...

The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a ...

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

A new treatment possibility has been confirmed for ischemic cardiovascular disease, which does not have a suitable treatment ...

The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...