Duchenne Muscular Dystrophy
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CLINICAL_TRIALS research · 2024-10-04Clinical Trial Update: phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
The phenotypeS study in non-ambulant Duchenne muscular dystrophy is currently recruiting, according to the trial update.
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CLINICAL_TRIALS research · 2024-09-23Clinical Trial Update: Wearable Technology to Assess Gait Function in SMA and DMD
A completed clinical trial evaluating wearable technology to assess gait function in spinal muscular atrophy type 3 and Duchenne muscular dy…
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CLINICAL_TRIALS trial_result · 2024-09-20Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy [Phase 1]
Phase 1 study evaluating PF-06939926 gene therapy in Duchenne muscular dystrophy was terminated. Results from the study have been posted.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) [Phase 1, Phase 2]
Open-label extension study of vesleteplirsen (SRP-5051) in Duchenne muscular dystrophy participants has terminated; results from the study w…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy [Phase 3]
This Phase 3 extension study evaluating casimersen or golodirsen in Duchenne Muscular Dystrophy has been terminated, and the results have be…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy [NA]
Pfizer-sponsored natural history study in Chinese male patients with Duchenne muscular dystrophy has completed, with results posted.
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CLINICAL_TRIALS research · 2024-09-03Clinical Trial Update: Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
A clinical trial update from University College London aims to define outcome measures for behavioral and emotional problems in dystrophinop…
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CLINICAL_TRIALS trial_result · 2024-08-30Clinical Trial Update: Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) [Phase 1]
A Phase 1 trial evaluating the safety and dosing of EN001 in patients with Duchenne Muscular Dystrophy has been completed.
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CLINICAL_TRIALS research · 2024-08-30Clinical Trial Update: Development of a Registry to Assess Natural History in Duchenne Muscular Dystrophy
This clinical trial update describes the development of a registry to characterize the natural history of Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS research · 2024-08-30Clinical Trial Update: Expanded Access Study of UC-MSC in DMD Patients
Expanded Access study update for UC-MSC therapy in Duchenne Muscular Dystrophy, with the study status listed as AVAILABLE.
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CLINICAL_TRIALS research · 2024-08-27Clinical Trial Update: Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
This clinical trial update characterizes skeletal and cardiac impairment in carriers of Duchenne muscular dystrophy and Becker muscular dyst…
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CLINICAL_TRIALS trial_result · 2024-08-26Clinical Trial Update: Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD [Phase 3]
A Phase 3 trial evaluating pamrevlumab plus systemic corticosteroids in ambulatory Duchenne muscular dystrophy was terminated, and trial res…
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CLINICAL_TRIALS trial_result · 2024-08-20Clinical Trial Update: Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) [Phase 2]
A Phase 2 study evaluating the safety, tolerability, and efficacy of viltolarsen in ambulant and non-ambulant boys with Duchenne muscular dy…
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CLINICAL_TRIALS other · 2024-08-20Clinical Trial Update: CureDuchenne Link®: A Resource for Research
CureDuchenne Link is currently recruiting for research related to Duchenne muscular dystrophy and Becker muscular dystrophy.
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CLINICAL_TRIALS research · 2024-08-06Clinical Trial Update: Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies [NA]
Clinical trial update describing a protocol to apply engagement pathways and resources for participation among people with Duchenne Muscular…
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CLINICAL_TRIALS trial_result · 2024-07-22Clinical Trial Update: Brain Involvement in Dystrophinopathies Part 2
A completed clinical trial update on brain involvement in dystrophinopathies, including Duchenne muscular dystrophy and Becker muscular dyst…
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CLINICAL_TRIALS trial_result · 2024-07-22Clinical Trial Update: Brain Involvement in Dystrophinopathies Part 1
Clinical trial update on brain involvement in dystrophinopathies, specifically Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (NC…
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CLINICAL_TRIALS research · 2024-07-16Clinical Trial Update: Effect of Captopril on GLS in Duchenne Myodystrophy [EARLY_Phase 1]
An early-phase (Phase 1) clinical trial is evaluating the effect of captopril on global longitudinal strain in Duchenne muscular dystrophy, …
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FDA approval · 2024-07-11FDA Approved: AMONDYS 45 — Supplemental Application (Sarepta Therapeutics, Inc.)
FDA approved a supplemental application for Amondys 45 (casimersen) from Sarepta Therapeutics, Inc., related to Duchenne muscular dystrophy.
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EMA withdrawal · 2024-06-28EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
The EMA recommends non-renewal of the marketing authorisation for Translarna (ataluren) for Duchenne muscular dystrophy in the EU after revi…
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FDA approval · 2024-06-05FDA Approved: AGAMREE — Supplemental Application (Catalyst Pharmaceuticals, Inc.)
FDA approved a supplemental application for AGAMREE (vamorolone) submitted by Catalyst Pharmaceuticals, Inc.
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FDA approval · 2024-06-05FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
The FDA approved a supplemental application for EMFLAZA (deflazacort) by PTC Therapeutics, Inc. This action updates the drug’s regulatory st…
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FDA approval · 2024-06-04FDA Approved: VYONDYS 53 — Supplemental Application (Sarepta Therapeutics, Inc.)
The FDA approved a supplemental application for VYONDYS 53 (golodirsen) filed by Sarepta Therapeutics, expanding regulatory status for Duche…
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CLINICAL_TRIALS trial_result · 2024-05-23Clinical Trial Update: A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 1, Phase 2]
Phase 1/2 safety study of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy completed, with results posted in May 2024.
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CLINICAL_TRIALS trial_result · 2024-05-13Clinical Trial Update: Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker. [NA]
Completed clinical trial assessing the effectiveness of a multimodal physiotherapy program using virtual reality glasses in individuals with…