Introducing Our Newest Trustee: Emma Simmonds “On behalf of the Board of Trustees at Action Duchenne, I extend a warm welcometo Emma as our newest …
Join our NEW Online Support Groups
We decided our regular online support groups needed a bit of a re-think. To make sure we’ve got everyone covered, there are now going to be 4 …
Action Duchenne are seeking Trustees (volunteer roles)
Action Duchenne are seeking Trustees (volunteer roles) Trustee Action Duchenne are currently searching for strong individuals to join the …
Action Duchenne are seeking Trustees (volunteer roles)Read More
Action Duchenne is seeking a Chairperson (volunteer role)
Action Duchenne is seeking a Chairperson (volunteer role) ChairpersonAction Duchenne are currently searching for a strong individual to guide and …
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NICE Publishes Draft Guidance on Vamorolone for the Treatment of Duchenne Muscular Dystrophy
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 …