The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...

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 ...

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 ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

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 ...

Regulators in the U.S. and the European Union agreed to review Biogen’s applications for approval of a higher dose Spinraza ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...

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