Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new therapeutic option for patients with the rare disease.

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular ... with symptoms usually first seen between two and five years of age. Symptoms worsen over time affecting the ability to ...

Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. At dosing, microdystrophin expression was 122.3% higher in a patient age three compared to ...