Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2020-04-15Clinical Trial Update: Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Clinical trial update on intramuscular rAAV1.CMV.huFollistatin344 for Duchenne muscular dystrophy completed with posted results.
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CLINICAL_TRIALS trial_result · 2020-04-07Clinical Trial Update: Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) [Phase 2]
Phase 2B study of ataluren (PTC124) in Duchenne/Becker muscular dystrophy completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-03-17Clinical Trial Update: Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Phase 1/2 trial investigating autologous bone marrow–derived stem cells for Duchenne muscular dystrophy; current status not provided.
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CLINICAL_TRIALS trial_result · 2020-01-02Clinical Trial Update: Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 proof-of-concept study evaluating activity and safety of ezutromid (SMT C1100) in boys with Duchenne Muscular Dystrophy was terminat…
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FDA approval · 2019-12-12FDA Approved: VYONDYS 53 — Original Application (Sarepta Therapeutics, Inc.)
The FDA approved Sarepta Therapeutics' golodirsen (VYONDYS 53) as the original application for treatment of Duchenne muscular dystrophy due …
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CLINICAL_TRIALS research · 2019-12-05Clinical Trial Update: Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Clinical trial update for antisense oligonucleotides in Duchenne Muscular Dystrophy (Phase 1/2) with completed status and results posted.
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CLINICAL_TRIALS trial_result · 2019-11-25Clinical Trial Update: An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy [Phase 2]
Open-label extension study assessing safety of PF-06252616 in boys with Duchenne Muscular Dystrophy; terminated with results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-09-24Clinical Trial Update: Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy [Phase 3]
Phase 3 clinical trial evaluating aldosterone inhibition as a therapeutic strategy for Duchenne Muscular Dystrophy has completed with result…
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CLINICAL_TRIALS trial_result · 2019-07-23Clinical Trial Update: An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Extension study to assess vamorolone in boys with Duchenne Muscular Dystrophy completed; results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-07-23Clinical Trial Update: A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy [Phase 2]
A Phase 2 trial of PF-06252616 in Duchenne Muscular Dystrophy was terminated; results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-07-18Clinical Trial Update: Rimeporide in Patients With Duchenne Muscular Dystrophy [Phase 1]
Phase 1 trial update for rimeporide in Duchenne muscular dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2019-07-10Clinical Trial Update: Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 rollover study evaluating efficacy, safety, and tolerability of eteplirsen in Duchenne Muscular Dystrophy completed with results pos…
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CLINICAL_TRIALS withdrawal · 2019-06-21Clinical Trial Update: A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) [Phase 3]
A Phase 3 trial of deflazacort (Emflaza) in Duchenne Muscular Dystrophy has been withdrawn.
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FDA approval · 2019-06-07FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
The FDA approved a supplemental application for EMFLAZA (deflazacort) submitted by PTC Therapeutics, Inc.
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FDA label_change · 2019-06-07FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
FDA approved a supplemental application for EMFLAZA (deflazacort) submitted by PTC Therapeutics, Inc. The approval updates the product label…
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CLINICAL_TRIALS trial_result · 2019-05-08Clinical Trial Update: Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD [Phase 1, Phase 2]
Phase 1/2 trial of RO7239361 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy terminated; results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-02-20Clinical Trial Update: Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy [Phase 2]
Phase 2 safety study of eteplirsen in advanced-stage Duchenne muscular dystrophy completed; results posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-02-08Clinical Trial Update: Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
A completed clinical trial from Benha University investigates quantitative muscle ultrasound as a progression marker in children with Duchen…
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CLINICAL_TRIALS trial_result · 2019-02-04Clinical Trial Update: Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
A completed clinical trial evaluated aerobic exercise in boys with Duchenne muscular dystrophy; the update notes the study status and summar…
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CLINICAL_TRIALS trial_result · 2019-01-28Clinical Trial Update: A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy [Phase 3]
This Phase 3 clinical trial update reports the completion of a study evaluating the efficacy and safety of GSK2402968 in Duchenne muscular d…
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CLINICAL_TRIALS trial_result · 2019-01-02Clinical Trial Update: A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 trial updating on vamorolone in boys with Duchenne muscular dystrophy has completed with results posted in 2019.
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FDA approval · 2018-10-11FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)
The FDA approved Sarepta Therapeutics' supplemental application for Exondys 51 (eteplirsen) for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2018-08-16Clinical Trial Update: Comparative Study of Strategies for Management of Duchenne Myopathy (DM) [Phase 4]
A Phase 4 comparative study evaluating management strategies for Duchenne-like myopathy is currently in an unknown status.
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CLINICAL_TRIALS trial_result · 2018-07-31Clinical Trial Update: Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
This clinical trial examined the relationship between muscle strength and hamstring flexibility in children with Duchenne muscular dystrophy…
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CLINICAL_TRIALS trial_result · 2018-06-15Clinical Trial Update: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies [Phase 2, Phase 3]
This update notes the completion of a Phase 2/Phase 3 clinical trial evaluating Coenzyme Q10 in combination with Lisinopril for muscular dys…