The symptoms of myotonic dystrophy may be obvious from birth or they can develop later -- during the teenage or adult years. Like other forms of muscular dystrophy, myotonic dystrophy leads to ...
What Is Duchenne Muscular Dystrophy? Muscular dystrophies are ... The disease almost always affects boys, and symptoms usually begin early in childhood. Children with DMD have a hard time standing ...
What Is Genetic Testing? Limb-Girdle Muscular Dystrophy Limb-girdle muscular dystrophy is a form of MD that develops in teenagers or young adults. Symptoms first become apparent in the hips, then 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 ...
The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...
An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines Agency (EMA) considerate it as an alternative than the current lower ...
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) 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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback