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 · 2026-08-17Clinical Trial Update: A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） [Phase 3]
Phase 3 trial update for TAS-205 in Duchenne muscular dystrophy has completed for REACH-DMD study.
-
EMA approval · 2026-07-03EMA Authorised: Duvyzat (givinostat hydrochloride monohydrate) — Italfarmaco S.p.A.
EMA authorised Duvyzat (givinostat hydrochloride monohydrate) for Duchenne muscular dystrophy under conditional and orphan designation.
-
CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Update: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy [Phase 3]
Phase 3 study of Deramiocel (CAP-1002) in ambulatory and non-ambulatory Duchenne muscular dystrophy patients is active but not recruiting.
-
CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients? [NA]
A clinical trial is evaluating external vagus nerve stimulation to reduce systemic inflammatory mediators in Duchenne muscular dystrophy; en…
-
CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy [Phase 4]
Phase 4 study evaluating long-term safety and effectiveness of vamorolone in boys with Duchenne Muscular Dystrophy; current status is active…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
A clinical trial update describes development of non-invasive prenatal diagnosis for single-gene disorders. The study, based in Paris, is cu…
-
CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback [NA]
A completed clinical trial assessed the feasibility of virtual reality in children with neuromuscular diseases and evaluated the effectivene…
-
CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101 [Phase 2]
This clinical trial update notes an open-label, long-term follow-up study of SAT-3247 for Duchenne Muscular Dystrophy, including participant…
-
CLINICAL_TRIALS other · 2026-04-23Clinical Trial Update: NS-050/NCNP-03 in Boys With DMD (Meteor50) [Phase 1, Phase 2]
Phase 1/2 trial NS-050/NCNP-03 for Duchenne muscular dystrophy is currently active but not recruiting.
-
CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting [Phase 4]
Phase 4 clinical trial update evaluating the safety and effectiveness of Sarepta's gene therapy Elevidys for Duchenne Muscular Dystrophy in …
-
CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Invasive Home Ventilation in Denmark
A Danish clinical trial sponsored by Rigshospitalet is studying invasive home ventilation for patients with neuromuscular diseases, includin…
-
CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: DMD- Interactive Virtual Reality Study [NA]
A completed clinical trial evaluating an interactive virtual reality study for Duchenne Muscular Dystrophy.
-
CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) [Phase 1]
Phase 1 clinical trial evaluating the safety and biodistribution of a single intrathecal dose of INS1201 in ambulatory male Duchenne Muscula…
-
CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders [NA]
A terminated clinical trial update investigating gut peptides and bone remodeling in children with neuromuscular disorders, sponsored by the…
-
CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This clinical trial update notes a real-world study evaluating Givinostat in patients with Duchenne muscular dystrophy, with the study curre…
-
CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients [Phase 2]
This document reports an update on a Phase 2 clinical trial of SAT-3247 for Duchenne muscular dystrophy in pediatric ambulatory patients, wh…
-
CLINICAL_TRIALS trial_result · 2026-04-09Clinical Trial Update: A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) [Phase 2]
This Phase 2 clinical trial update indicates that satralizumab is being studied for efficacy and safety in Duchenne muscular dystrophy, with…
-
CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants [Phase 1]
This is a Phase 1 trial evaluating the safety and pharmacokinetics of GRT6019 in healthy male participants for Duchenne muscular dystrophy; …
-
CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
A clinical trial update describing a study to establish walking-related digital biomarkers in rare childhood-onset progressive neuromuscular…
-
EMA approval · 2026-04-08EMA Authorised: Agamree (vamorolone) — Santhera Pharmaceuticals (Deutschland) GmbH
The European Medicines Agency authorised Agamree (vamorolone) by Santhera Pharmaceuticals for Duchenne muscular dystrophy, with orphan medic…
-
CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of BMN 351 in participants with Du…
-
CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
This clinical trial update describes the ongoing managed access program for Del-zota in participants with Duchenne muscular dystrophy mutati…
-
CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Urinary Titin Biomarker in DMD [NA]
Clinical trial update regarding a urinary titin biomarker study in Duchenne muscular dystrophy and Becker muscular dystrophy, currently recr…
-
CLINICAL_TRIALS research · 2026-04-07Clinical Trial Update: DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
A clinical trial is recruiting to investigate DMD gene variants and related cardiac dysfunction in young males with dystrophinopathies, incl…
-
CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 1]
This Phase 1 clinical trial is evaluating the safety and expression of delandistrogene moxeparvovec (SRP-9001) in participants with Duchenne…