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 trial_result · 2025-02-03Clinical Trial Update: A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy [Phase 2]
The Phase 2 study of ATL1102 vs placebo in non-ambulatory Duchenne Muscular Dystrophy has been terminated.
-
CLINICAL_TRIALS trial_result · 2025-01-14Clinical Trial Update: Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
A clinical trial status update regarding follow-up care with home noninvasive ventilation for patients with Duchenne muscular dystrophy.
-
CLINICAL_TRIALS trial_result · 2025-01-09Clinical Trial Update: HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) [Phase 2]
A Phase 2 HOPE-Duchenne trial evaluating CAP-1002 for Duchenne Muscular Dystrophy–related cardiomyopathy has completed and results were post…
-
CLINICAL_TRIALS trial_result · 2025-01-09Clinical Trial Update: Stacking Exercises Aid the Decline in FVC and Sick Time [Phase 4]
A Phase 4 clinical trial in Duchenne Muscular Dystrophy evaluated stacking exercises and their impact on forced vital capacity decline and s…
-
CLINICAL_TRIALS trial_result · 2024-12-11Clinical Trial Update: Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) [Phase 3]
Phase 3 RACER53 trial results for Viltepso (viltolarsen) in ambulant boys with Duchenne Muscular Dystrophy have been posted, reporting effic…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2024-12-10Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 study evaluating delandistrogene moxeparvovec (SRP-9001) for Duchenne muscular dystrophy has completed and results have been posted.
-
CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) [Phase 1]
A Phase 1 study evaluating ASP0367 (MA-0211) in pediatric male participants with Duchenne Muscular Dystrophy was terminated.
-
CLINICAL_TRIALS trial_result · 2024-09-20Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy [Phase 1]
Phase 1 study evaluating PF-06939926 gene therapy in Duchenne muscular dystrophy was terminated. Results from the study have been posted.
-
CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) [Phase 1, Phase 2]
Open-label extension study of vesleteplirsen (SRP-5051) in Duchenne muscular dystrophy participants has terminated; results from the study w…
-
CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy [Phase 3]
This Phase 3 extension study evaluating casimersen or golodirsen in Duchenne Muscular Dystrophy has been terminated, and the results have be…
-
CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy [NA]
Pfizer-sponsored natural history study in Chinese male patients with Duchenne muscular dystrophy has completed, with results posted.
-
CLINICAL_TRIALS trial_result · 2024-08-30Clinical Trial Update: Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) [Phase 1]
A Phase 1 trial evaluating the safety and dosing of EN001 in patients with Duchenne Muscular Dystrophy has been completed.
-
CLINICAL_TRIALS trial_result · 2024-08-26Clinical Trial Update: Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD [Phase 3]
A Phase 3 trial evaluating pamrevlumab plus systemic corticosteroids in ambulatory Duchenne muscular dystrophy was terminated, and trial res…
-
CLINICAL_TRIALS trial_result · 2024-08-20Clinical Trial Update: Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) [Phase 2]
A Phase 2 study evaluating the safety, tolerability, and efficacy of viltolarsen in ambulant and non-ambulant boys with Duchenne muscular dy…
-
CLINICAL_TRIALS trial_result · 2024-07-22Clinical Trial Update: Brain Involvement in Dystrophinopathies Part 2
A completed clinical trial update on brain involvement in dystrophinopathies, including Duchenne muscular dystrophy and Becker muscular dyst…
-
CLINICAL_TRIALS trial_result · 2024-07-22Clinical Trial Update: Brain Involvement in Dystrophinopathies Part 1
Clinical trial update on brain involvement in dystrophinopathies, specifically Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (NC…
-
CLINICAL_TRIALS trial_result · 2024-05-23Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 1, Phase 2]
Phase 1/2 safety study of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy completed, with results posted in May 2024.
-
CLINICAL_TRIALS trial_result · 2024-05-13Clinical Trial Update: Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker. [NA]
Completed clinical trial assessing the effectiveness of a multimodal physiotherapy program using virtual reality glasses in individuals with…
-
CLINICAL_TRIALS trial_result · 2024-04-25Clinical Trial Update: Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
A clinical trial evaluating upper-extremity muscle strength, balance, and functional skills in Duchenne Muscular Dystrophy has completed. Th…
-
CLINICAL_TRIALS trial_result · 2024-04-24Clinical Trial Update: Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) [Phase 2]
Phase 2 trial update for HOPE-OLE evaluating cardiomyopathy progression in Duchenne Muscular Dystrophy; study completed with results posted.
-
CLINICAL_TRIALS trial_result · 2024-04-01Clinical Trial Update: Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A Phase 1/2a clinical trial of SQY51 in pediatric and adult patients with Duchenne Muscular Dystrophy is underway to assess safety, pharmaco…
-
CLINICAL_TRIALS trial_result · 2024-04-01Clinical Trial Update: A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 2]
Phase 2 clinical trial evaluating safety and pharmacokinetics of ataluren in young children with nonsense mutation Duchenne muscular dystrop…
-
CLINICAL_TRIALS trial_result · 2024-03-25Clinical Trial Update: Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Clinical trial update evaluating EN001 for Duchenne Muscular Dystrophy across Phase 1 and Phase 2.
-
CLINICAL_TRIALS trial_result · 2024-03-12Clinical Trial Update: Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 trial evaluating pamrevlumab vs placebo with systemic corticosteroids in non-ambulatory Duchenne Muscular Dystrophy was terminated; …
-
CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: Study of DS-5141b in Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Phase 1/2 study of DS-5141b in Duchenne Muscular Dystrophy has completed; results posted in 2024.