The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...

We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...

Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for ...

constipation electrolyte imbalances kidney problems loss of balance Read on to learn more about why older adults are more prone to dehydration, the symptoms to look out for, and what you can do to ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...

Spasticity, dystonia, tremor and migraine were variably present; cognitive impairment was severe in early childhood cases, but was absent in adults. In contrast to previous reports, two of the ...

Especially, when you consider that it was able to report positive results from its phase 2 CANYON study, using its drug sevasemten for the treatment of patients with Becker Muscular Dystrophy [BMD ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). Published in the December issue of the Journal of Biological Chemistry, researchers characterize the ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

after seeing positive results in pediatric and young adult patients with spinal muscular atrophy (SMA). The Phase III STEER study met its primary endpoint of an increase in Hammersmith Functional ...