On the Monday the 29th of January, Sarepta Therapeutics announced positive data from Part B of the MOMENTUM study. The MOMENTUM Study is a global …
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) …
Dyne Therapeutics Announces Positive Initial Clinical Data From DELIVER Trials in DMD Patients
Dyne Therapeutics have reported their initial clinical data from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are …
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient …
Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study
Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study On the 15th of December 2023, Wave Life Sciences announced the initiation …
<strong>Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study </strong>Read More
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 …
FDA Grants Edgewise Therapeutics Inc Orphan Drug and Rare Paediatric Disease Designations for Its Muscular Dystrophy Program.
On the 30th November Edgewise Therapeutics Inc. (a leading muscle disease biopharmaceutical company) announced that the U.S. Food & Drug …
United Nations Officially Designates September 7th as World Duchenne Awareness Day
The global rare disease community is celebrating the UN’s first formal acknowledgement of a day dedicated to a rare disease. In a groundbreaking …
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 Solid Biosciences Inc., a leading life …
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 authorisation PTC Therapeutics' Translarna, a critical …
Thank you for attending the Action Duchenne Annual International Conference 2023!
Welcome to the Annual Action Duchenne International Conference 2023
Welcome to the Annual Action Duchenne International Conference 2023 On behalf of the whole team at Action Duchenne, I would like to give you the …
Welcome to the Annual Action Duchenne International Conference 2023Read More
