Duchenne Muscular Dystrophy
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy [Phase 3]
This clinical trial update reports that Capricor's Phase 3 study of Deramiocel (CAP-1002) in Duchenne muscular dystrophy is currently active…
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 2, Phase 3]
A status update that RGX-202 gene therapy for Duchenne Muscular Dystrophy is in Phase 2/3 and currently recruiting.
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CLINICAL_TRIALS research · 2025-12-16Clinical Trial Update: Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening [NA]
An ongoing newborn-screening study evaluating decentralized monitoring of motor development in children with spinal muscular atrophy or Duch…
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CLINICAL_TRIALS research · 2025-12-15Clinical Trial Update: Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) [Phase 1, Phase 2]
An open-label Phase 1/2 study (FORWARD-53) of WVE-N531 in Duchenne Muscular Dystrophy is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-15Clinical Trial Update: The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
The Baby Duchenne Study is a pediatric clinical trial evaluating developmental and clinical outcomes during the first three years of life in…
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FDA approval · 2025-12-12FDA Approved: DEFLAZACORT — Original Application (Amneal Pharmaceuticals NY LLC)
FDA approved Amneal Pharmaceuticals NY LLC's original application for a generic version of deflazacort.
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CLINICAL_TRIALS research · 2025-12-04Clinical Trial Update: A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy. [Phase 1, Phase 2]
A clinical trial update describes a Phase 1/2 study to evaluate safety, tolerability, and efficacy of BBM-D101 in Duchenne muscular dystroph…
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CLINICAL_TRIALS other · 2025-12-03Clinical Trial Update: The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder [NA]
A clinical trial evaluating a muscle-mimicking fabric-type shoulder orthosis to improve upper-limb movements in patients with neuromuscular …
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CLINICAL_TRIALS research · 2025-12-02Clinical Trial Update: An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
An observational study comparing Delandistrogene Moxeparvovec with standard of care in Duchenne muscular dystrophy is currently enrolling by…
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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.
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CLINICAL_TRIALS research · 2025-11-25Clinical Trial Update: AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This clinical trial update describes the AFFINITY BEYOND study, an anti-AAV8 antibody assessment in males with Duchenne muscular dystrophy, …
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
A clinical trial at Massachusetts General Hospital is recruiting to study extracellular RNA biomarkers in Duchenne Muscular Dystrophy. The s…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Biomarker Development for Muscular Dystrophies
Massachusetts General Hospital sponsors a clinical trial update focused on developing biomarkers for muscular dystrophy. The study is curren…
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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.
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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…
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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…
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CLINICAL_TRIALS withdrawal · 2025-11-20Clinical Trial Update: Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) [NA]
The clinical trial NCT05558813 studying the natural history of Duchenne muscular dystrophy-related cardiomyopathy has been withdrawn.
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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 …
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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 …
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy [Phase 2]
Phase 2 study of EDG-5506 in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy. The trial is cu…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim
A longitudinal clinical trial update from Vanderbilt University Medical Center describes using wearable technology to monitor hyperglycemia …
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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.
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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.
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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…
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EMA withdrawal · 2025-10-20EMA Refused: Elevidys (delandistrogene moxeparvovec) — Roche Registration GmbH
The European Medicines Agency refused to grant marketing authorization for Elevidys (delandistrogene moxeparvovec) from Roche Registration G…