The American Journal of Managed Care9 天
Exploring Care Needs, Physical Activity in Myotonic Dystrophy
Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...
BioPharma Dive2 天
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a disorder on the radars of several drugmakers.
The American Journal of Managed Care10 天
How Access to SMA Treatment Varies Globally and by Insurance Type
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...
MedPage Today11 天
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Science Daily11 天
Restoring heart function in Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...