Duchenne Muscular Dystrophy
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Sign up freeTop drugs studied for this disease
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FDA approval · 2021-02-25FDA Approved: AMONDYS 45 — Original Application (Sarepta Therapeutics, Inc.)
FDA approves AMONDYS 45 (casimersen) via an original application for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2021-02-21Clinical Trial Update: A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of MNK-1411 in Duchenne Muscular Dystrophy was terminated; results posted.
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FDA approval · 2021-02-11FDA Approved: VYONDYS 53 — Supplemental Application (Sarepta Therapeutics, Inc.)
FDA approved a supplemental application for VYONDYS 53 (golodirsen) from Sarepta Therapeutics, Inc. The approval pertains to Duchenne muscul…
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CLINICAL_TRIALS trial_result · 2021-01-29Clinical Trial Update: Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients [Phase 1, Phase 2]
Phase 1/2 clinical trial investigating transplantation of myoblasts in Duchenne Muscular Dystrophy patients.
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CLINICAL_TRIALS trial_result · 2021-01-20Clinical Trial Update: Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A safety and efficacy study of IGF-1 in Duchenne Muscular Dystrophy completed phases 1 and 2.
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EMA withdrawal · 2021-01-18EMA Application withdrawn: Puldysa (idebenone;titanium dioxide) — Santhera Pharmaceuticals (Deutschland) GmbH
EMA withdrawn the application for Puldysa (idebenone; titanium dioxide) by Santhera for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2020-12-21Clinical Trial Update: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy [Phase 2, Phase 3]
Phase 2/3 trial evaluating RO7239361 in ambulatory boys with Duchenne muscular dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-11-25Clinical Trial Update: Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada [Phase 3]
Phase 3 trial update for ataluren in previously treated nmDBMD participants with nonsense mutation Duchenne/Becker muscular dystrophy; study…
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CLINICAL_TRIALS trial_result · 2020-11-23Clinical Trial Update: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy [Phase 3]
Open-label extension study update for edasalonexent in boys with Duchenne muscular dystrophy; Phase 3 trial terminated.
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CLINICAL_TRIALS trial_result · 2020-10-29Clinical Trial Update: Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2a extension study of ataluren (PTC124) in Duchenne Muscular Dystrophy was terminated; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Flu Vaccine Study in Neuromuscular Patients 2011 [Phase 4]
A Phase 4 clinical trial update on a flu vaccine study in pediatric neuromuscular disease patients has completed, with results posted in 202…
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Phase I/II Study of SRP-4053 in DMD Patients [Phase 1, Phase 2]
Phase I/II study update for SRP-4053 in Duchenne Muscular Dystrophy completed with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-08-28Clinical Trial Update: Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of ataluren in young boys with Duchenne muscular dystrophy completed; results posted in 2020.
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FDA approval · 2020-08-12FDA Approved: VILTEPSO — Original Application (NS Pharma, Inc.)
The FDA approved Viltepso (viltolarsen) from NS Pharma, Inc. under Original NDA212154 for Duchenne muscular dystrophy amenable to exon 53 sk…
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CLINICAL_TRIALS trial_result · 2020-08-11Clinical Trial Update: An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy [Phase 3]
Phase 3 extension study for ataluren (PTC124) in nonsense mutation dystrophinopathy was terminated; results were posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-08-04Clinical Trial Update: Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 3]
Phase 3 trial of ataluren in nonsense mutation Duchenne muscular dystrophy completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-07-29Clinical Trial Update: Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) [Phase 2]
Phase 2 trial update for Ataluren (PTC124) in nonambulatory Duchenne/Becker muscular dystrophy due to nonsense mutations; study terminated w…
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CLINICAL_TRIALS trial_result · 2020-07-22Clinical Trial Update: Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy [Phase 2]
Phase 2 safety study of eteplirsen in early-stage Duchenne Muscular Dystrophy completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-07-15Clinical Trial Update: Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) [Phase 2]
Phase 2B extension study of ataluren in Duchenne/Becker muscular dystrophy was terminated; results posted in 2020.
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FDA approval · 2020-07-10FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
The FDA approved a supplemental application for EMFLAZA (deflazacort) submitted by PTC Therapeutics, Inc.
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FDA approval · 2020-07-10FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
FDA approved a supplemental application for EMFLAZA (deflazacort) from PTC Therapeutics, expanding/confirming its labeling for Duchenne musc…
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FDA approval · 2020-07-07FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)
The FDA approved a supplemental application for Exondys 51 (eteplirsen) from Sarepta Therapeutics, Inc.
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CLINICAL_TRIALS trial_result · 2020-07-01Clinical Trial Update: Study of Eteplirsen in DMD Patients [Phase 3]
Phase 3 trial evaluating eteplirsen in Duchenne Muscular Dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-06-23Clinical Trial Update: A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD [Phase 1, Phase 2]
A completed Phase 1/2 trial assessed safety, tolerability, pharmacokinetics, histology effects, and clinical parameters of givinostat in chi…
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CLINICAL_TRIALS trial_result · 2020-04-20Clinical Trial Update: A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy [Phase 2]
Phase IIa study of TAS-205 for Duchenne Muscular Dystrophy has completed; results posted in 2020.