Duchenne Muscular Dystrophy
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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…
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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…
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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…
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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…
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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…
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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…
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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; …
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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…
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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…
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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…
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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…
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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…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children
A multicenter cohort study protocol investigating Duchenne muscular dystrophy and Becker muscular dystrophy in Western Chinese children. The…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD) [Phase 1]
Phase 1 clinical trial evaluating safety, tolerability, and initial efficacy of a single intravenous infusion of JWK007 in Duchenne Muscular…
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy [NA]
A study is evaluating the safety and tolerability of Rituxan (rituximab) in Duchenne Muscular Dystrophy. The trial is not yet recruiting.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) [Phase 1, Phase 2]
Clinical trial update on PBGENE-DMD for Duchenne muscular dystrophy, outlining a Phase 1/2a safety and preliminary efficacy study that is no…
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: Duchenne Muscular Dystrophy Video Assessment Registry
This clinical trial update reports completion of the Duchenne Muscular Dystrophy Video Assessment Registry and does not provide efficacy or …
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CLINICAL_TRIALS research · 2026-03-09Clinical Trial Update: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating the safety and potential efficacy of ENTR-601-45 for Duchenne muscular dystrophy that is amenable t…
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
NS Pharma's NS-089/NCNP-02-201 is a Phase 2 trial for Duchenne muscular dystrophy in boys with exon 44 mutations, and recruitment is ongoing…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: Trial of Cell Based Therapy for DMD [Phase 1]
Phase 1 clinical trial update for a cell-based therapy in Duchenne muscular dystrophy, currently recruiting.
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) [Phase 3]
Phase 3 clinical trial update for SGT-003 gene therapy in ambulant males with Duchenne Muscular Dystrophy; the study is currently recruiting…
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD [Phase 1, Phase 2]
Solid Biosciences is running a Phase 1/2 microdystrophin gene transfer study in children and adolescents with Duchenne muscular dystrophy; t…
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) [Phase 1, Phase 2]
Solid Biosciences is conducting the INSPIRE DUCHENNE study of SGT-003 gene therapy for Duchenne muscular dystrophy. The Phase 1/2 trial is c…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Relation Between Muscle Architecture and Functional Ability in Children With Duchenne Muscular Dystrophy
A completed clinical study investigating how muscle architecture relates to functional ability in children with Duchenne muscular dystrophy.
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Effects of Posture and Quality of Life in Duchenne Muscular Dystrophy Children [NA]
A recruiting clinical trial sponsored by Riphah International University aims to examine how posture influences quality of life in children …