Recommendation is based on Phase 3 EPIDYS trial data that demonstrated Duvyzat (givinostat) provides statistically and ...

The EC has granted orphan drug designation to Dyne Therapeutics’ DYNE-251 for treating Duchenne muscular dystrophy (DMD).

A Shotts mum will head a 45-strong group taking part in this weekend’s Glasgow Kiltwalk to raise money for her four-year-old ...

The orphan drug designation is particularly noteworthy as it can provide Dyne with a range of benefits, including reduced regulatory fees, assistance with clinical protocols, eligibility for research ...

Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for ...

Families say it's 'completely frustrating' that children with Duchenne Muscular Dystrophy are being denied access to free ...

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as ...

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as well as some cancers.

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as well as some cancers.In ...

Jordan McLinn has been an advocate since childhood to add add Duchenne muscular dystrophy to Indiana’s newborn screening ...

Over time, this can lead to cavities and gum disease, so flossing is a must!” Michelle Jorgensen, doctor of dental surgery ...

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...