CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...

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 ...

EMA re-confirms non-renewal of conditional marketing authorisation of Duchenne muscular dystrophy medicine, Translarna: Amsterdam, The Netherlands Monday, October 21, 2024, 10:00 ...

NICE's HST committee has three more technology evaluations underway already, including ataluren for Duchenne Muscular Dystrophy. The first drug approved through the HST process was Alexion's ...

The drug's active substance, ataluren, helps cells bypass the mutation to produce functional dystrophin protein. Following ...

PTCT announced positive results on two different long-term extension studies on vatiquinone.The company also announced ...

Seaport Therapeutics Inc. has followed up its recent fundraiser with an oversubscribed $225 million series B financing that will help set it on the path to a phase IIb study of major depressive ...

Passed Up for the Ticket, Shapiro Tries to Deliver Pennsylvania for Harris Gov. Josh Shapiro of Pennsylvania may not be on the verge of the vice presidency, but he says he has everything riding on ...

Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory ...

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

Opinion to be reviewed by European Commission - WARREN, N.J., Oct. 18, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ) ann ...