PTC Therapeutics has announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a negative …
This is the latest news about the development and use of Translarna for Duchenne muscular dystrophy. Translarna can treat approximately 10-15% of case of Duchenne – those that are caused by a nonsense mutation.
There’s also lots of information and videos explaining these potential treatments in our information pages.
MHRA Translarna Survey
IntroductionIn 2014, the European Medicines Agency (EMA), granted a ‘Conditional Marketing Authorisation’ to Translarna for the treatment of patients …
PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
PTC Therapeutics has announced today that the European Commission (EC) has decided not to adopt the European Medicine Agency (EMA) Committee for …
PTC Community Statement on Translarna (Ataluren) Market Authorisation in the European Union and Great Britain
PTC has provided a community statement regarding the recently decision by the CHMP/EMA to not renew the marketing authorisation for Translarna …
The European Medicines Agency confirms recommendation for non-renewal of authorisation in the EU of Duchenne muscular dystrophy medicine Translarna
We shared challenging news with the Duchenne community in September 2023. The European Medicine Agency's (EMA) human medicines committee (CHMP) …
PTC Therapeutics Provides Updates on Translarna™ (ataluren) regulatory activities in Europe and the United States
PTC Therapeutics has announced, on the 5th of December 2023, an update on their regulatory progress with the European Medicines Association (EMA) and …
Stakes ‘couldn’t be higher’ – PTC CEO on propect of Translarna losing EU conditional authorisation
Stakes 'couldn't be higher' – PTC CEO on prospect of Translarna losing EU conditional authorisationPTC Therapeutics' Translarna, a critical …
Action Duchenne Expresses Deep Concern Over CHMP’s Negative Opinion on Translarna™ for Duchenne Muscular Dystrophy
Action Duchenne Expresses Deep Concern Over CHMP's Negative Opinion on Translarna™ for Duchenne Muscular DystrophyAction Duchenne expresses profound …
BREAKING NEWS
NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment TranslarnaNICE has published final guidance recommending …
NICE publishes draft guidance on access to Duchenne treatment Translarna
NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there …
NICE publishes draft guidance on access to Duchenne treatment TranslarnaRead More
Translarna update
The treatment ataluren (Translarna) for Duchenne muscular dystrophy has now exited managed access and entered the NICE re-evaluation process. In order …
Translarna accepted by Scottish Medicines Consortium
A drug called Translarna can help treat an underlying condition of Duchenne muscular dystrophyAction Duchenne, Duchenne Family Support Group, Muscular …
Translarna accepted by Scottish Medicines ConsortiumRead More
