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

Kedrion is proud to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...

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

Kedrion Biopharma receives US FDA orphan drug designation for plasma-derived treatment for congenital aceruloplasminemia: Forte Lee, New Jersey Saturday, August 9, 2025, 17:00 Hrs ...

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

In April 2025, the FDA granted orphan drug designation to spesolimab-sbzo for the treatment of pyoderma gangrenosum. This ...

The cystinosis market is poised for growth amid increasing newborn screening, innovative cysteamine formulations, and potential gene therapy breakthroughs. As a rare metabolic disorder, cystinosis is ...

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

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

Starlight Therapeutics, a wholly owned subsidiary of Lantern Pharma Inc. (NASDAQ: LTRN), today announced that the U.S. Food ...

At the low end, the drugs include $119,000 a year to treat certain rare genetic disorders. At the high end: a gene therapy ...

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 ...