T O APPRECIATE the dilemmas the Food and Drug Administration (FDA) faces when it reviews new drugs targeting rare diseases, ...

How could Trump's demands that drugmakers lower U.S. prices or face penalties impact pharma and biotech stocks? Seeking Alpha ...

The FDA has denied Stealth BioTherapeutics’ request to reconsider its complete response letter, leaving the fate of the biotech and its investigational treatment for an ultrarare genetic disease ...

April 2, 2025 Quoin Pharmaceuticals announced positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study. After six months of treatment with QRX003, significant ...

Precision is advancing the final U.S. investigational new drug (IND)-enabling toxicology studies with an anticipated IND and/or clinical trial application (CTA) filing targeted by the end of 2025 with ...

How political red tape and a drug company’s thirst for profits limited the reach of a drug that experts believe could have ...

Developed by regenerative nanomedicine pioneer Samuel I. Stupp, the therapy harnesses molecular motion to reverse paralysis ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...