We stand with the Duchenne community in expressing our disappointment regarding the National Institute for Health and Care Excellence's (NICE) recent …
FDA Approves Givinostat for the Treatment of Duchenne Muscular Dystrophy in the USA
Further to our previous article on the publication of the phase 3 EPIDYS trial positive results for Givinostat, a histone deacetylase (HDAC) inhibitor …
FDA Approves Givinostat for the Treatment of Duchenne Muscular Dystrophy in the USARead More
Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology
We are thrilled to announce exciting new developments regarding the investigational drug Givinostat, after Italfarmaco S.p.A. announced today that the …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical Trial
PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical TrialRead More
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 …
Annual General Meeting 2024
Annual General Meeting 2024 The Trustees of Action Duchenne would like to express their gratitude for the continued support from the Duchenne …
Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication
Sarepta Therapeutics, Inc, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the company's ‘Efficacy Supplement’ to …
Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (Duchenne)
On February 13th, Edgewise Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for …
Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
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 …
