The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...

Caleb Kasner and his brother Duncan Kasner move around a little differently than average kids. The two brothers have Duchenne ...

Alfie, from Newry, County Down, was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years old. His dad Jamie Pentony said the new drug, Givinostat, could slow hi ...

Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of ...

Evrysdi may affect a man’s ability to have children ... spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne ...

Robert Davidson is the first UK patient recruited to a new medical trial looking to support children with heart failure ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

The Nijmegen diagnostic criteria uses a numerical scale (range 0 to 12) for the number of clinical (muscular, CNS and selected ... Table 1 lists the signs and symptoms of CRPS-I in each of the ...

Leah Messer is opening up about navigating her daughter Ali's muscular dystrophy as the 15-year-old takes a big step in her life: high school. In an interview with TooFab ahead of the new season ...

Bronx Rep. Ritchie Torres has accused Gov. Kathy Hochul of bungling oversight of New York’s early intervention program for toddlers with developmental disabilities such as autism and Down syndrome.

2025 could be a breakout year for the Duchenne muscular dystrophy space. Although several therapies ... Additionally, the company has been generating data in children under 4 years of age with hopes ...