Millions of people around the world live with rare diseases, most of them caused by a misstep in their genes. Some conditions ...

Friday a group of local childhood friends embarked on a journey down the Rogue River, hoping to get to all the way to the ...

A Southern Oregon family is embarking on a remarkable journey to support their son, who is facing a life-threatening disease.

Insmed invests heavily in a robust pipeline with multi-billion potential, positioning for future growth. Check out why INSM ...

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 ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

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.