The landscape of rare diseases is constantly changing as new ailments and conditions are identified and reported regularly.

The FDA established its accelerated approval program in 1992 to allow drugs to reach the market quickly for life-threatening ...

Of the 24 drugs, three approvals deviated from the others -- aducanumab; the synthetic hormone hydroxyprogesterone caproate ...

NICE’s positive final guidance for Agamree follows confirmation that no appeals were submitted against its positive December ...

Oppenheimer adjusted its price target on Keros Therapeutics (NASDAQ:KROS) shares, reducing it significantly to $23.00 from ...

An Irish woman has launched a fundraiser to help raise money for medical treatment for her seven-year-old son. Una Ennis' son ...

Duchenne muscular dystrophy (DMD), a rare condition in females, causes muscle weakness. 27-year-old Stephanie, who was ...

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...

The OIG report points to gaps in documentation and decision-making transparency, particularly in the case of Aduhelm, where FDA records indicated only 13 meetings with Biogen, but congressional ...

Undervalued biotech company Regenxbio Inc. shows promise with cutting-edge gene therapy technology for AMD and MPS II. Click ...

A trial team led by Latham’s Mike Morin and David Frazier and Finnegan’s William (Bill) Raich helped Sarepta Therapeutics ...