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
April 1, 2024 by Lizzie Deeble
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
April 1, 2024 by Lizzie Deeble
Action Duchenne is seeking a Chairperson (volunteer role) ChairpersonAction Duchenne are currently searching for a strong individual to guide and …
Action Duchenne is seeking a Chairperson (volunteer role)Read More
March 28, 2024 by John Marrin
We stand with the Duchenne community in expressing our disappointment regarding the National Institute for Health and Care Excellence's (NICE) recent …
March 22, 2024 by John Marrin
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
March 21, 2024 by John Marrin
We are thrilled to announce exciting new developments regarding the investigational drug Givinostat, after Italfarmaco S.p.A. announced today that the …
March 5, 2024 by John Marrin
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
February 29, 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 …
February 23, 2024 by Lizzie Deeble
Annual General Meeting 2024 The Trustees of Action Duchenne would like to express their gratitude for the continued support from the Duchenne …
February 16, 2024 by John Marrin
Sarepta Therapeutics, Inc, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the company's ‘Efficacy Supplement’ to …
February 15, 2024 by John Marrin
On February 13th, Edgewise Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for …
January 31, 2024 by John Marrin
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 …
January 27, 2024 by Sofiya Got
We shared challenging news with the Duchenne community in September 2023. The European Medicine Agency's (EMA) human medicines committee (CHMP) …
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