The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

The Supreme Court today (Friday) while hearing a plea by a medical candidate suffering from muscular dystrophy observed that ...

Benitec Biopharma Inc.'s positive results for BB-301 in OPMD treatment show promise, but potential risks and financial ...

On Friday 11 October, seven comedians – led by Jon Richardson – came together to entertain an audience of over 1,000 people at Aylesbury Waterside Theatre and raise money for charity.

The three-horse race to treat a devastating form of muscular dystrophy is now down to two. One of the key players, Avidity ...

SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion on the renewal ...

On 29 September, Tony Dale, 80, began an 80-mile walk from Cambridge back to his hometown, planning to walk the distance in ...

PTC Therapeutics (PTCT) announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has ...

On Friday 11 October, seven comedians – led by Jon Richardson – came together to entertain an audience of over 1,000 people ...

Two young brothers are getting a life-changing medical treatment all due to the University of New Mexico Hospital. The 10 and ...

ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced today that the first participant has been ...