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 · 2026-02-24Clinical Trial Update: Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Registry update shows that the Translarna (ataluren) study in nonsense mutation Duchenne muscular dystrophy has completed.
-
CLINICAL_TRIALS research · 2026-02-23Clinical Trial Update: Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy
This clinical trial from Lokman Hekim University is investigating the relationship between executive functions and occupational performance …
-
CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: A Remote Study Using Technology to Assess Outcomes in DMD
The trial update describes a remote, technology-enabled study to assess outcomes in Duchenne Muscular Dystrophy. Recruitment has not yet beg…
-
CLINICAL_TRIALS trial_result · 2026-02-17Clinical Trial Update: An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE) [EARLY_Phase 1]
An open-label, multi-dose Phase 1 study evaluated the safety and potential long-lasting effects of a CRISPR gene-editing therapy in Duchenne…
-
CLINICAL_TRIALS trial_result · 2026-02-13Clinical Trial Update: A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants [Phase 1]
Grünenthal GmbH completed a Phase 1 study to evaluate the safety, tolerability, and pharmacokinetics of GRT6019 in healthy male participants…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: Natural History of Duchenne Muscular Dystrophy
This entry describes an ongoing clinical trial studying the natural history of Duchenne Muscular Dystrophy, sponsored by Genethon, which is …
-
CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: Registry for Duchenne and Becker Muscular Dystrophy
This entry documents a registry study for Duchenne and Becker muscular dystrophy led by Dr. Andreas Ziegler, currently not yet recruiting.
-
CLINICAL_TRIALS research · 2026-02-10Clinical Trial Update: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) [Phase 2]
This clinical trial update shows Roche's delandistrogene moxeparvovec being studied in children under four with Duchenne Muscular Dystrophy …
-
CLINICAL_TRIALS trial_result · 2026-02-06Clinical Trial Update: A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study [Phase 3]
This update presents long-term follow-up data from participants who previously received delandistrogene moxeparvovec (SRP-9001) in a Duchenn…
-
CLINICAL_TRIALS research · 2026-01-30Clinical Trial Update: Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
A clinical trial update describes multiparametric ultrafast ultrasound biomarkers being studied for Duchenne muscular dystrophy and Becker m…
-
CLINICAL_TRIALS research · 2026-01-29Clinical Trial Update: Factors Influencing Physical Activity Levels in Children With Duchenne Muscular Dystrophy: An ICF-CY-Based Study
This update reports a clinical trial evaluating factors that influence physical activity levels in children with Duchenne muscular dystrophy…
-
CLINICAL_TRIALS research · 2026-01-27Clinical Trial Update: Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy [NA]
A clinical trial update shows that a gamified occupational therapy program is recruiting adolescents with Duchenne Muscular Dystrophy.
-
CLINICAL_TRIALS research · 2026-01-27Clinical Trial Update: The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial [NA]
This clinical trial is enrolling by invitation to study whether a dual-tasking program improves cognitive and physical function and independ…
-
CLINICAL_TRIALS research · 2026-01-27Clinical Trial Update: DMD and Gamified Physiotherapy [NA]
The document reports an update on a Duchenne Muscular Dystrophy trial investigating gamified physiotherapy. The study is currently not yet r…
-
FDA approval · 2026-01-27FDA Approved: DEFLAZACORT ORAL SUSPENSION — Original Application (Sun Pharmaceutical Industries, Inc.)
FDA approves a generic deflazacort oral suspension from Sun Pharmaceutical Industries under ANDA 219930.
-
CLINICAL_TRIALS trial_result · 2026-01-26Clinical Trial Update: Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 clinical trial update for oral ifetroban in Duchenne muscular dystrophy patients, including those with cardiomyopathy, completed wit…
-
CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA [NA]
This clinical trial update describes a study to enhance self-efficacy in caregivers of children with Duchenne Muscular Dystrophy and Spinal …
-
CLINICAL_TRIALS research · 2026-01-21Clinical Trial Update: Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study [Phase 2, Phase 3]
This clinical trial update reports a phase 2/3 long-term safety and tolerability study of givinostat for Duchenne muscular dystrophy, curren…
-
CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
This update covers an Expanded Access Protocol for boys with Duchenne Muscular Dystrophy sponsored by Santhera Pharmaceuticals.
-
CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy [EARLY_Phase 1]
A pediatric Phase 1 study evaluating safety and tolerability of the investigational drug RAG-18 in Duchenne muscular dystrophy is active but…
-
CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Swiss Registry for Neuromuscular Disorders
The University of Bern is coordinating a Swiss registry for neuromuscular disorders with ongoing recruitment, covering multiple conditions i…
-
CLINICAL_TRIALS trial_result · 2026-01-14Clinical Trial Update: Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD [NA]
A completed clinical trial from Cairo University evaluating the effects of virtual reality and whole body vibration on muscle strength and b…
-
FDA approval · 2026-01-13FDA Approved: DEFLAZACORT ORAL SUSPENSION — Supplemental Application (Zydus Pharmaceuticals (USA) Inc.)
The FDA approved a supplemental application to add an oral suspension formulation of deflazacort, expanding the available dosage form for Zy…
-
FDA approval · 2026-01-06FDA Approved: AMONDYS 45 — Supplemental Application (Sarepta Therapeutics, Inc.)
FDA approved a supplemental application for Amondys 45 (casimersen) from Sarepta Therapeutics, Inc.
-
CLINICAL_TRIALS research · 2025-12-24Clinical Trial Update: Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy [Phase 1]
Phase 1 clinical trial assessing SGLT2 inhibitors as therapy for Duchenne cardiomyopathy in Duchenne muscular dystrophy; recruitment not yet…