Duchenne Muscular Dystrophy
📧 Sign up to get email alerts when something new happens for Duchenne Muscular Dystrophy.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS withdrawal · 2025-11-20Clinical Trial Update: Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) [NA]
The clinical trial NCT05558813 studying the natural history of Duchenne muscular dystrophy-related cardiomyopathy has been withdrawn.
-
EMA withdrawal · 2025-10-20EMA Refused: Elevidys (delandistrogene moxeparvovec) — Roche Registration GmbH
The European Medicines Agency refused to grant marketing authorization for Elevidys (delandistrogene moxeparvovec) from Roche Registration G…
-
CLINICAL_TRIALS withdrawal · 2025-09-04Clinical Trial Update: A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 [Phase 1]
This clinical trial update reports that Sarepta Therapeutics' gene transfer therapy Delandistrogene moxeparvovec (SRP-9001) for Duchenne Mus…
-
CLINICAL_TRIALS withdrawal · 2025-06-24Clinical Trial Update: A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment [Phase 2]
A Phase 2 study of PGN-EDO51 or placebo for Duchenne muscular dystrophy amenable to exon 51 skipping has been withdrawn.
-
EMA withdrawal · 2024-10-29EMA Expired: Translarna (ataluren) — PTC Therapeutics International Limited
EMA status indicates that Translarna (ataluren) has expired for Duchenne muscular dystrophy as of 28 October 2024. The marketing authorizati…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
EMA withdrawal · 2024-10-18Translarna: EMA re-confirms non-renewal of authorisation of Duchenne muscular dystrophy medicine
The EMA confirms that it will not renew Translarna's authorization for Duchenne muscular dystrophy after a re-examination, because effective…
-
EMA withdrawal · 2024-06-28EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
The EMA recommends non-renewal of the marketing authorisation for Translarna (ataluren) for Duchenne muscular dystrophy in the EU after revi…
-
EMA withdrawal · 2024-01-26EMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA confirms recommendation to non-renew the marketing authorisation for Translarna, a medicine for Duchenne muscular dystrophy.
-
EMA withdrawal · 2023-09-15EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA recommends non-renewal of authorisation for Translarna (ataluren) for Duchenne muscular dystrophy, effectively ending its marketing auth…
-
EMA withdrawal · 2021-01-18EMA Application withdrawn: Puldysa (idebenone;titanium dioxide) — Santhera Pharmaceuticals (Deutschland) GmbH
EMA withdrawn the application for Puldysa (idebenone; titanium dioxide) by Santhera for Duchenne muscular dystrophy.
-
CLINICAL_TRIALS withdrawal · 2019-06-21Clinical Trial Update: A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
A Phase 3 trial of deflazacort (Emflaza) in Duchenne Muscular Dystrophy has been withdrawn.
-
CLINICAL_TRIALS withdrawal · 2018-05-14Clinical Trial Update: Relationship Between PFTs and Pdi in DMD [NA]
A clinical trial evaluating the relationship between pulmonary function tests and transdiaphragmatic pressure in Duchenne muscular dystrophy…
-
CLINICAL_TRIALS withdrawal · 2017-12-08Clinical Trial Update: A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) [Phase 1, Phase 2]
Phase I/II study of BMN053 for Duchenne muscular dystrophy has been terminated.
-
EMA withdrawal · 2016-09-06EMA Application withdrawn: Kyndrisa — BioMarin International Limited
The EMA withdrew the application for Kyndrisa for Duchenne muscular dystrophy.
-
CLINICAL_TRIALS withdrawal · 2015-02-18Clinical Trial Update: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy [Phase 2]
A Phase 2 trial evaluating chronotherapy of corticosteroids in Duchenne muscular dystrophy has been withdrawn.
-
CLINICAL_TRIALS withdrawal · 2015-02-04Clinical Trial Update: Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
A clinical trial investigating test-retest reliability of pulmonary function tests in patients with Duchenne muscular dystrophy has been wit…
-
CLINICAL_TRIALS withdrawal · 2014-03-24Clinical Trial Update: Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol [Phase 3]
The Phase 3 clinical trial of Drisapersen for Duchenne muscular dystrophy has been withdrawn by sponsor GlaxoSmithKline.