A lightweight robotic knee device improves strength, muscle growth, and standing ability in children with SMA, with benefits ...

As families continue a month-long protest outside the government administration building, parents of children with Duchenne muscular dystrophy met with ...

Dyne has aligned with the U.S. Food and Drug Administration (FDA) on the FORZETTO Phase 3 trial design and protocol. FORZETTO ...

Strong Buy on DM1 & DMD clinical progress, BLA timeline, and strong cash runway through 2028. Click for this DYN update.

Both prosecutors and Mackenzie Shirilla have made different cases as to why she slammed her car into a brick wall and killed ...

Semaglutide and colchicine may help prevent atrial fibrillation by protecting heart tissue, reducing inflammation, and ...

The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, ...

Natalie has seen her body and speech deteriorate; she has lost her independence and dignity — but she says new research into ...

The US has ramped up economic pressure on Cuba – was that just the first step in a potential ‘friendly takeover’? (Photo: ...

The FDA has introduced models intended to accelerate rare disease drug development, but recent reversals of guidance from the ...

Dyno Therapeutics launches two new AAV Capsids, AI platform for rare disease therapeutic development: Watertown, Massachusetts Saturday, May 16, 2026, 13:00 Hrs [IST] Dyno Therape ...

As FDA activity accelerates and big pharma searches for its next generation of blockbuster drugs, select biotech companies ...