Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with Friedreich ataxia, a rare neurodegenerative disorder BRAVE study will explore the ...

A controversial proposal to change the Inflation Reduction Act’s pricing framework for rare disease drugs has been dropped ...

Illegal THC on every corner, Illegal grow ops in plain sight, Yet the DEA continues to block MMJ BioPharma Cultivation's FDA sanctioned application to produce pharmaceutical-grade cannabis pending ...

Shares of Cero Therapeutics Holdings drew increased retail investor chatter on Tuesday after the company said that the FDA ...

Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of the one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing ...

The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

ABOUT ORPHAN DRUG DESIGNATION The FDA grants Orphan Drug Designation to investigational drugs intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. Benefits ...

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

Discover six kidney disease companies actively working on innovative treatments that could make a difference to patients' lives.

Following a complete response letter issued last October over CMC issues, CSL Behring LLC gained U.S. FDA approval June 16 of its humanized anti-factor XIIa monoclonal antibody, garadacimab (CSL-312), ...