This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024. Following this, Santhera has already started launch ...

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

REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS ...

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 ...

AbbVie and Gilead are going back to their roots and leaning on their established areas of expertise to set themselves up for ...

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 ...

DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...

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

Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after ...