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 · 2025-06-22Clinical 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 research · 2025-06-13Clinical Trial Update: ML-Based Multi-Sensor Fall Risk Screening in DMD
Clinical trial update on a machine-learning based multi-sensor fall risk screening tool for Duchenne muscular dystrophy; the study is curren…
-
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 research · 2025-06-06Clinical Trial Update: Follow-up Study on Female Carriers With DMD Gene Variants
Clinical trial update describing a follow-up study enrolling female carriers of DMD gene variants to assess clinical characteristics related…
-
CLINICAL_TRIALS research · 2025-05-20Clinical Trial Update: Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study [NA]
A clinical trial at Centre Hospitalier Universitaire de Liege assesses muscular pathology in patients and controls under the ActiLiège Next …
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
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 research · 2025-05-14Clinical Trial Update: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping [Phase 2]
A Phase 2 open-label study evaluating AOC-1044 for Duchenne muscular dystrophy with mutations amenable to exon 44 skipping is currently acti…
-
CLINICAL_TRIALS research · 2025-05-11Clinical Trial Update: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy [Phase 3]
A Phase 3 trial evaluating the efficacy, safety and tolerability of givinostat in non-ambulant Duchenne muscular dystrophy patients; current…
-
CLINICAL_TRIALS research · 2025-04-30Clinical Trial Update: DMD Voice: Qualitative Interviews With Patients and Caregivers
A ClinicalTrials.gov entry update describes a recruiting qualitative study called 'DMD Voice' that interviews patients with Duchenne Muscula…
-
EMA approval · 2025-04-25New treatment against Duchenne muscular dystrophy
The European Medicines Agency announced the approval of Duvyzat as a new treatment for Duchenne muscular dystrophy, for patients aged six an…
-
CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
-
CLINICAL_TRIALS research · 2025-04-02Clinical Trial Update: Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy [EARLY_Phase 1]
Early-phase clinical trial update on LE051 evaluating safety, tolerability, and potential efficacy in Duchenne Muscular Dystrophy; the study…
-
CLINICAL_TRIALS research · 2025-04-01Clinical Trial Update: Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies. [NA]
A clinical trial update evaluating the effectiveness of a 5-week digital respiratory practice program in children with Duchenne muscular dys…
-
CLINICAL_TRIALS research · 2025-03-28Clinical Trial Update: Psychoeducation Program for Parents of Children with Duchenne Muscular Dystrophy [NA]
This clinical trial update covers a psychoeducation program for parents of children with Duchenne muscular dystrophy, with participant recru…
-
CLINICAL_TRIALS research · 2025-03-25Clinical Trial Update: Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy [EARLY_Phase 1]
An early-phase clinical trial is evaluating the safety and potential efficacy of BBM-D101 for Duchenne muscular dystrophy. The study is curr…
-
CLINICAL_TRIALS research · 2025-03-21Clinical Trial Update: NSAA NON-Interventional Study Protocol
This document provides an update on a non-interventional study protocol using NSAA for Duchenne muscular dystrophy and notes that the study …
-
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 research · 2025-03-13Clinical Trial Update: Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
A clinical trial update notes a NOT_YET_RECRUITING study examining social cognition in Duchenne muscular dystrophy, Becker muscular dystroph…
-
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 research · 2025-03-06Clinical Trial Update: Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
This ClinicalTrials.gov study from Assistance Publique - Hôpitaux de Paris in France aims to estimate non-reimbursable costs for patients wi…
-
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 research · 2025-03-05Clinical Trial Update: Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy
This clinical trial update reports on the physical activity level of Norwegian boys with Duchenne Muscular Dystrophy; the study status is co…
-
CLINICAL_TRIALS research · 2025-02-27Clinical Trial Update: Tadalafil as Adjuvant Therapy for DMD [Phase 2, Phase 3]
Clinical trial update indicating tadalafil is being studied as adjuvant therapy for Duchenne Muscular Dystrophy in Phase 2/3 trials, with ac…
-
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…