Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2025-02-24Clinical Trial Update: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of CAP-1002 in ambulatory and non-ambulatory Duchenne muscular dystrophy completed, with results posted.
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CLINICAL_TRIALS trial_result · 2025-02-24Clinical Trial Update: Open-label Extension of the HOPE-2 Trial [Phase 2]
Open-label extension update for Capricor’s HOPE-2 Phase 2 trial in Duchenne muscular dystrophy, with results posted in February 2025.
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CLINICAL_TRIALS research · 2025-02-18Clinical Trial Update: Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases [NA]
A recruiting clinical trial using transcriptomic analysis to reduce misdiagnosis in patients with rare genetic muscle diseases, including Du…
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CLINICAL_TRIALS research · 2025-02-10Clinical Trial Update: The Duchenne Registry
The Duchenne Registry trial is currently recruiting, sponsored by The Duchenne Registry, and targets Duchenne muscular dystrophy and Becker …
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CLINICAL_TRIALS trial_result · 2025-02-03Clinical Trial Update: A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy [Phase 2]
The Phase 2 study of ATL1102 vs placebo in non-ambulatory Duchenne Muscular Dystrophy has been terminated.
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CLINICAL_TRIALS research · 2025-01-30Clinical Trial Update: Hydrotherapy in Duchenne Muscular Dystrophy (DMD) [NA]
This clinical trial update describes hydrotherapy for Duchenne Muscular Dystrophy, with the study currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2025-01-14Clinical Trial Update: Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
A clinical trial status update regarding follow-up care with home noninvasive ventilation for patients with Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2025-01-09Clinical Trial Update: HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) [Phase 2]
A Phase 2 HOPE-Duchenne trial evaluating CAP-1002 for Duchenne Muscular Dystrophy–related cardiomyopathy has completed and results were post…
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CLINICAL_TRIALS trial_result · 2025-01-09Clinical Trial Update: Stacking Exercises Aid the Decline in FVC and Sick Time [Phase 4]
A Phase 4 clinical trial in Duchenne Muscular Dystrophy evaluated stacking exercises and their impact on forced vital capacity decline and s…
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CLINICAL_TRIALS research · 2025-01-03Clinical Trial Update: Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
A pediatric neuromuscular disease trial is recruiting to evaluate respiratory function, thoracoabdominal movements, and exercise capacity.
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CLINICAL_TRIALS research · 2025-01-01Clinical Trial Update: Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
A clinical trial is examining the relationships among scoliosis, pain, quality of life, and trunk muscle compensation in Duchenne muscular d…
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FDA approval · 2024-12-27FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)
The FDA approved a supplemental application for Exondys 51 (eteplirsen) from Sarepta Therapeutics.
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CLINICAL_TRIALS research · 2024-12-27Clinical Trial Update: Analysis of Motor Performance in Children with Duchene's Muscular Dystrophy
A clinical trial update from Cairo University describes planned analysis of motor performance in children with Duchenne muscular dystrophy, …
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CLINICAL_TRIALS research · 2024-12-16Clinical Trial Update: Pediatric Radio Frequency Coils Generic
Clinical trial update indicating an ongoing pediatric radio frequency coils generic study with status ACTIVE_NOT_RECRUITING.
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CLINICAL_TRIALS research · 2024-12-13Clinical Trial Update: Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy
Clinical trial update evaluating brain dysfunction in patients with Duchenne Muscular Dystrophy; the study is currently not yet recruiting.
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CLINICAL_TRIALS trial_result · 2024-12-11Clinical Trial Update: Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) [Phase 3]
Phase 3 RACER53 trial results for Viltepso (viltolarsen) in ambulant boys with Duchenne Muscular Dystrophy have been posted, reporting effic…
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CLINICAL_TRIALS trial_result · 2024-12-10Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 study evaluating delandistrogene moxeparvovec (SRP-9001) for Duchenne muscular dystrophy has completed and results have been posted.
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CLINICAL_TRIALS other · 2024-12-02Clinical Trial Update: The U.K. NorthStar Clinical Network
The U.K. NorthStar Clinical Network trial for Duchenne Muscular Dystrophy is not yet recruiting, according to the update.
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CLINICAL_TRIALS research · 2024-11-14Clinical Trial Update: FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a longitudinal epidemiology study by xCures to follow outcomes across multiple rare diseases, and the study is currently recruitin…
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FDA approval · 2024-11-05FDA Approved: DEFLAZACORT — Original Application (Upsher-Smith Laboratories, LLC)
FDA approved the original Abbreviated New Drug Application for Deflazacort from Upsher-Smith Laboratories, granting a generic version of def…
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FDA approval · 2024-10-29FDA Approved: DEFLAZACORT ORAL SUSPENSION — Supplemental Application (Cranbury Pharmaceuticals, LLC)
The FDA approved a supplemental application for Deflazacort oral suspension by Cranbury Pharmaceuticals, expanding the availability of defla…
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EMA withdrawal · 2024-10-29EMA Expired: Translarna (ataluren) — PTC Therapeutics International Limited
EMA status indicates that Translarna (ataluren) has expired for Duchenne muscular dystrophy as of 28 October 2024. The marketing authorizati…
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) [Phase 1]
A Phase 1 study evaluating ASP0367 (MA-0211) in pediatric male participants with Duchenne Muscular Dystrophy was terminated.
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EMA withdrawal · 2024-10-18Translarna: EMA re-confirms non-renewal of authorisation of Duchenne muscular dystrophy medicine
The EMA confirms that it will not renew Translarna's authorization for Duchenne muscular dystrophy after a re-examination, because effective…
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CLINICAL_TRIALS research · 2024-10-16Clinical Trial Update: Effects of Lumbopelvic Stabilization-based Physiotherapy and Rehabilitation Training in Duchenne Muscular Dystrophy [NA]
This clinical trial examines a lumbopelvic stabilization-based physiotherapy and rehabilitation program for Duchenne muscular dystrophy, wit…