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 ...

Leerink Partners analyst Joseph Schwartz has maintained their bullish stance on SLDB stock, giving a Buy rating yesterday.Stay Ahead of the ...

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 ...

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 ...

Roll Call reports that the newly finalized rule will allow for health care providers who have not seen a patient in person to prescribe six months’ worth of buprenorphine via telehealth. Also in ...

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

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...