People living with a rare neurological degenerative disease have called on the Government to make the first treatment for the ...

A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of ...

A Senate hearing probed whether the Food and Drug Administration's process for approving rare-disease therapies is ...

Larimar's stock jumps 55% in a month after the FDA grants breakthrough therapy status to its FA drug nomlabofusp and backs a potential accelerated path.

Larimar Therapeutics Inc LRMR shares are trading sharply higher Tuesday after the company said the U.S. Food and Drug ...

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the agency’s bureaucracy affects innovation, with one witness testifying that ...

VY1706 clinical entry and VY7523 clinical data anticipated H2 2026 - - Validating brain-targeted capsids in humans: expect ...

The back-and-forth between the FDA and gene therapy developer uniQure is getting messier, with an agency spokesperson now denying the biotech’s claims about a sham trial request for the rare ...

Biogen Inc. (NASDAQ: BIIB) on Friday reported better-than-expected fourth-quarter earnings, with adjusted EPS of $1.99, beating the consensus of $1.59. The Alzheimer's drug maker reported revenues of ...

FDA clearance of IND application for VO659 with plans to initiate US-based clinical trial later this yearNew data highlight distinctive mechanism of action of exon 1–directed CAG repeat–targeting ASO ...

One biotech firm raised more than $100 million and another generated more than $200 million in separate stock sales after their valuations soared on positive news for their lead drug candidates.