Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

Matilda Baptiste is 16 from Hall Green needs help to buy vital equipment to help her live independently with Duchenne ...

It was, on the outside looking in, a marvellous Christmas for the Ennis household in Tallaght. Archie (7) was totally excited ...

The company says it has in total “nearly 3,000 people at 11 locations in Maine, Connecticut, California, Japan and China, and ...

Wheeling around the gym, always smiling, always chatting it up with players, Brandon Wechsler brought a charm and positivity ...

An accident and a malfunction render both of columnist Betty Vertin's accessible vans useless, leaving the family without transportation.

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

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 ...

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 ...