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 · 2025-12-02Clinical Trial Update: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment [Phase 2]
PepGen's Phase 2 study of PGN-EDO51 for Duchenne Muscular Dystrophy amenable to exon 51 skipping has been terminated.
-
CLINICAL_TRIALS trial_result · 2025-11-21Clinical Trial Update: Nutritional Status and Therapy in DMD Patients [NA]
A clinical trial evaluating nutritional status and therapy in Duchenne muscular dystrophy patients has completed.
-
CLINICAL_TRIALS trial_result · 2025-11-20Clinical Trial Update: Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy [Phase 3]
Phase 3 clinical trial evaluating nebivolol for prevention of left ventricular systolic dysfunction in Duchenne muscular dystrophy has compl…
-
CLINICAL_TRIALS trial_result · 2025-11-20Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) [Phase 1]
A Phase 1 trial evaluating Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy in participants with pre-existing antibod…
-
CLINICAL_TRIALS trial_result · 2025-11-18Clinical Trial Update: Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 studies evaluating Sarepta's Casimersen (SRP-4045) and Golodirsen (SRP-4053) in Duchenne Muscular Dystrophy have completed; results …
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2025-11-12Clinical Trial Update: A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) [Phase 2]
This clinical trial update reports on the Phase 2 study of EDG-5506 for Duchenne Muscular Dystrophy in children. The trial is currently not …
-
CLINICAL_TRIALS trial_result · 2025-10-24Clinical Trial Update: A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 trial of vamorolone in boys aged 2 to <4 and 7 to <18 with Duchenne Muscular Dystrophy completed; results have been posted.
-
CLINICAL_TRIALS trial_result · 2025-10-21Clinical Trial Update: Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy [Phase 2]
Pfizer's Phase 2 study of Fordadistrogene Movaparvovec for early-stage Duchenne muscular dystrophy has been terminated.
-
CLINICAL_TRIALS trial_result · 2025-10-21Clinical Trial Update: A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. [Phase 3]
A Phase 3 study evaluating a Pfizer experimental gene therapy for Duchenne Muscular Dystrophy has been terminated. The trial aimed to assess…
-
CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study [NA]
This completed randomized controlled trial evaluated the effects of functional inspiratory muscle training with tele-rehabilitation on respi…
-
CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. [Phase 1, Phase 2]
Phase 1/2 clinical trial of an AAV9 U7snRNA gene therapy for Duchenne muscular dystrophy caused by exon 2 duplications has completed, with r…
-
CLINICAL_TRIALS trial_result · 2025-08-27Clinical Trial Update: IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère [NA]
A completed clinical trial evaluating free-breathing cardiac MRI in patients with Duchenne muscular dystrophy or Becker muscular dystrophy.
-
CLINICAL_TRIALS trial_result · 2025-08-15Clinical Trial Update: Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age [Phase 2]
Phase 2 clinical trial protocol repurposing empagliflozin for Duchenne muscular dystrophy–associated dilated cardiomyopathy in children aged…
-
CLINICAL_TRIALS trial_result · 2025-08-14Clinical Trial Update: Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
A completed clinical trial conducted by Virginia Commonwealth University assessed neurodevelopmental needs in Duchenne muscular dystrophy.
-
CLINICAL_TRIALS trial_result · 2025-07-14Clinical Trial Update: Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping [Phase 1, Phase 2]
Completed Phase 1/2 clinical trial of AOC 1044 in healthy adult volunteers and Duchenne muscular dystrophy patients with exon 44 skipping am…
-
CLINICAL_TRIALS trial_result · 2025-06-22Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 study update for delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy, reporting ongoing evaluation of safety and …
-
CLINICAL_TRIALS trial_result · 2025-06-08Clinical Trial Update: Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy [Phase 3]
Phase 3 study evaluating safety and efficacy of PF-06939926 for Duchenne Muscular Dystrophy has posted results; the trial is currently activ…
-
CLINICAL_TRIALS trial_result · 2025-05-15Clinical Trial Update: First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort [EARLY_Phase 1]
First-in-human SAD/MAD safety and pharmacokinetic study of an investigational therapy for Duchenne muscular dystrophy by Satellos Bioscience…
-
CLINICAL_TRIALS trial_result · 2025-03-20Clinical Trial Update: Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
This document reports an update on a completed clinical trial studying cardiac involvement in patients with Duchenne muscular dystrophy and …
-
CLINICAL_TRIALS trial_result · 2025-03-10Clinical Trial Update: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment [Phase 2]
A Phase 2 two-part study evaluating dose determination and subsequent expansion for vesleteplirsen (SRP-5051) in Duchenne muscular dystrophy…
-
CLINICAL_TRIALS trial_result · 2025-03-07Clinical Trial Update: Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy [NA]
Completed clinical trial studying a sleep intervention in young boys with Duchenne Muscular Dystrophy. The study was led by the University o…
-
CLINICAL_TRIALS trial_result · 2025-03-05Clinical Trial Update: Regular Physical Exercise in Duchenne Muscular Dystrophy
The clinical trial evaluating the impact of regular physical exercise in Duchenne muscular dystrophy has completed.
-
CLINICAL_TRIALS trial_result · 2025-02-27Clinical Trial Update: Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy [Phase 1]
Phase 1 clinical trial assessing modulation of SERCA2a to improve intra-myocytic calcium trafficking in Duchenne muscular dystrophy-associat…
-
CLINICAL_TRIALS trial_result · 2025-02-24Clinical Trial Update: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of CAP-1002 in ambulatory and non-ambulatory Duchenne muscular dystrophy completed, with results posted.
-
CLINICAL_TRIALS trial_result · 2025-02-24Clinical Trial Update: Open-label Extension of the HOPE-2 Trial [Phase 2]
Open-label extension update for Capricor’s HOPE-2 Phase 2 trial in Duchenne muscular dystrophy, with results posted in February 2025.