Breakthrough Therapy Designation granted to nomlabofusp for the treatment of adults and children with FA based on FDA’s review of available clinical data from open label studyContinued alignment with ...

Jason Brady ended up in the hospital around fifty times by the time he turned two years old, and he was officially diagnosed ...

How, 16, from Liverpool, was diagnosed with Friedreich's Ataxia in 2025 - a life-limiting disorder that progressively damages the nervous system.

Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher diseaseParis, March 18, 2026. The US Food and Drug ...

He ended up in the hospital around 50 times by the time he turned two and was officially diagnosed at age seven ...

US FDA grants Breakthrough Therapy designation to Sanofi’s venglustat for type 3 Gaucher disease: Paris Sanofi announced that the US Fo ...

Zevra Therapeutics ZVRA shares are up on Tuesday following a new partnership with GeneDx Holdings Corp. WGS to enhance genetic testing for Niemann-Pick Disease type C, or NPC. Niemann-Pick disease ...

Solid Biosciences (SLDB) stock retains a "Strong Buy" rating, driven by robust interim data for SGT-003 in pediatric Duchenne Muscular Dystrophy [DMD]. Read the full analysis here.

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the ...

Expanded label for once-yearly parasiticide treatment for dogs now includes Haemaphysalis longicornis (Asian longhorned tick) and Amblyomma maculatum (Gulf Coast tick) for 12 monthsRAHWAY, ...

Shares of Larimar Therapeutics LRMR rose 55% in the past month after the company announced key regulatory updates related to nomlabofusp, its lead pipeline asset. Larimar outlined upcoming milestones ...