The Scottish Medicines Consortium (SMC) has completed a reassessment of Translarna (ataluren) and has decided not to recommend it for routine use …
Translarna (Ataluren) Not Accepted for Continued Use Under NHSScotlandRead More
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.
8 June 2026 by John Marrin
The Scottish Medicines Consortium (SMC) has completed a reassessment of Translarna (ataluren) and has decided not to recommend it for routine use …
Translarna (Ataluren) Not Accepted for Continued Use Under NHSScotlandRead More

15 April 2025 by John Marrin
The European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency …

5 July 2024 by John Marrin
PTC Therapeutics has announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a negative …
22 May 2024 by Lizzie Cox
Introduction In 2014, the European Medicines Agency (EMA), granted a ‘Conditional Marketing Authorisation’ to Translarna for the treatment of …
20 May 2024 by Lizzie Cox
PTC Therapeutics has announced today that the European Commission (EC) has decided not to adopt the European Medicine Agency (EMA) Committee for …

29 February 2024 by John Marrin
PTC has provided a community statement regarding the recently decision by the CHMP/EMA to not renew the marketing authorisation for Translarna …

27 January 2024 by Lizzie Cox
We shared challenging news with the Duchenne community in September 2023. The European Medicine Agency's (EMA) human medicines committee (CHMP) …

10 December 2023 by Lizzie Cox
PTC Therapeutics has announced, on the 5th of December 2023, an update on their regulatory progress with the European Medicines Association (EMA) and …

16 November 2023 by Lizzie Cox
Stakes 'couldn't be higher' – PTC CEO on prospect of Translarna losing EU conditional authorisation PTC Therapeutics' Translarna, a critical …
15 September 2023 by Lizzie Cox
Action Duchenne Expresses Deep Concern Over CHMP's Negative Opinion on Translarna™ for Duchenne Muscular Dystrophy Action Duchenne expresses …

19 January 2023 by Lizzie Cox
NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment Translarna NICE has published final guidance …

30 September 2022 by Lynnette
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
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