A first-in-human clinical trial evaluating an investigational gene therapy targeting complement-mediated pathways in ...

Lindsey Vonn is back on her feet after a wicked crash at the Olympics nearly cost her a leg. However, she suffered from ...

Scholar Rock Holding has resubmitted its application for its spinal muscular atrophy treatment to the Food and Drug Administration. The biopharmaceutical company said that it has sent in a new ...

Discusses BLA Resubmission for Apitegromab in Spinal Muscular Atrophy and Regulatory Progress March 31, 2026 8:00 ...

Now to a major leap in the medical care of babies born with a devastating genetic condition. A diagnosis of type one spinal ...

MDA’s research funding for Dr. Adrian Krainer’s research led to the groundbreaking discovery of SPINRAZA®. FDA Approves High ...

Under the terms of a definitive agreement, Biogen will acquire Apellis for $41 per share in cash, or approximately $5.6 ...

The approval was anchored on data from the pivotal DEVOTE study that investigated the efficacy and safety of the High Dose ...

Advancing Zervimesine (CT1812) towards late-stage trials for dementia with Lewy bodies (DLB) psychosis Fully funded $80 million Phase 2 START trial readout expected in the second half of 2027 PURCHASE ...

In a phase 2 trial, OCU410, a modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration, ...

Scholar Rock (NASDAQ:SRRK) said it has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug ...

Kevin Rodriguez Zavala, 32, was found unresponsive after riding the Stardust Racers roller coaster at Universal Orlando's ...