Certain proteins implicated in immune response and the body's process to stop bleeding may be biomarkers of BMD progression, ...

The first few months of 2025 brought hope to the rare disease community. Food and Drug Administration leadership promised ...

Insmed’s (INSM) treatment of Duchenne muscular dystrophy, the single-stranded, non-replicating, recombinant adeno-associated virus serotype 9 ...