Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2024-04-25Clinical Trial Update: Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
A clinical trial evaluating upper-extremity muscle strength, balance, and functional skills in Duchenne Muscular Dystrophy has completed. Th…
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FDA approval · 2024-04-25FDA Approved: DEFLAZACORT ORAL SUSPENSION — Original Application (Cranbury Pharmaceuticals, LLC)
FDA approved Deflazacort Oral Suspension from Cranbury Pharmaceuticals as an Original Application (ANDA), providing a generic version of def…
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CLINICAL_TRIALS trial_result · 2024-04-24Clinical Trial Update: Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) [Phase 2]
Phase 2 trial update for HOPE-OLE evaluating cardiomyopathy progression in Duchenne Muscular Dystrophy; study completed with results posted.
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CLINICAL_TRIALS trial_result · 2024-04-01Clinical Trial Update: Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A Phase 1/2a clinical trial of SQY51 in pediatric and adult patients with Duchenne Muscular Dystrophy is underway to assess safety, pharmaco…
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CLINICAL_TRIALS trial_result · 2024-04-01Clinical Trial Update: A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 2]
Phase 2 clinical trial evaluating safety and pharmacokinetics of ataluren in young children with nonsense mutation Duchenne muscular dystrop…
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CLINICAL_TRIALS trial_result · 2024-03-25Clinical Trial Update: Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Clinical trial update evaluating EN001 for Duchenne Muscular Dystrophy across Phase 1 and Phase 2.
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CLINICAL_TRIALS trial_result · 2024-03-12Clinical Trial Update: Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) [Phase 3]
Phase 3 trial evaluating pamrevlumab vs placebo with systemic corticosteroids in non-ambulatory Duchenne Muscular Dystrophy was terminated; …
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: Study of DS-5141b in Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Phase 1/2 study of DS-5141b in Duchenne Muscular Dystrophy has completed; results posted in 2024.
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CLINICAL_TRIALS trial_result · 2024-02-14Clinical Trial Update: Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) [Phase 3]
Phase 3 trial update for viltolarsen in ambulant boys with Duchenne muscular dystrophy (DMD).
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FDA approval · 2024-02-09FDA Approved: DEFLAZACORT — Original Application (Aurobindo Pharma Limited)
FDA approved the original ANDA for deflazacort from Aurobindo Pharma Limited, enabling a generic version of deflazacort to be marketed in th…
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EMA withdrawal · 2024-01-26EMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA confirms recommendation to non-renew the marketing authorisation for Translarna, a medicine for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2024-01-23Clinical Trial Update: TXA127 in Non-Ambulant Patients With DMD Cardiomyopathy [Phase 2]
Phase 2 clinical trial update for TXA127 in non-ambulant Duchenne muscular dystrophy–associated cardiomyopathy.
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CLINICAL_TRIALS trial_result · 2024-01-11Clinical Trial Update: Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval [Phase 1]
Phase 1 trial assessing placebo-corrected QT/QTc effects of ITF2357 (Givinostat) at therapeutic and supratherapeutic doses alongside moxiflo…
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FDA approval · 2023-10-26FDA Approved: AGAMREE — Original Application (Catalyst Pharmaceuticals, Inc.)
FDA approved AGAMREE (vamorolone) from Catalyst Pharmaceuticals in an original NDA for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2023-10-23Clinical Trial Update: Spironolactone Versus Prednisolone in DMD [Phase 1]
Phase 1 trial comparing spironolactone versus prednisolone in Duchenne muscular dystrophy was terminated; results posted.
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CLINICAL_TRIALS trial_result · 2023-10-12Clinical Trial Update: A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. [Phase 2]
A 48-week Open Label study evaluated the efficacy and safety of AMONDYS 45, EXONDYS 51, and VYONDYS 53 in Duchenne muscular dystrophy patien…
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CLINICAL_TRIALS trial_result · 2023-09-21Clinical Trial Update: Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Pilot dose-escalation trial evaluating canakinumab (ILARIS) in Duchenne muscular dystrophy; Phase 1/2, status not specified.
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EMA withdrawal · 2023-09-15EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA recommends non-renewal of authorisation for Translarna (ataluren) for Duchenne muscular dystrophy, effectively ending its marketing auth…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) [Phase 1, Phase 2]
A randomized, double-blind, placebo-controlled Phase 1/2 study of delandistrogene moxeparvovec (SRP-9001) for Duchenne muscular dystrophy ha…
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CLINICAL_TRIALS trial_result · 2023-08-18Clinical Trial Update: A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) [Phase 2]
A Phase 2 trial evaluating safety, tolerability, and efficacy of eteplirsen in Duchenne Muscular Dystrophy participants who completed a prio…
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FDA approval · 2023-03-23FDA Approved: AMONDYS 45 — Supplemental Application (Sarepta Therapeutics, Inc.)
FDA approved a supplemental application for Amondys 45 (casimersen) from Sarepta Therapeutics, Inc.
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CLINICAL_TRIALS trial_result · 2023-02-02Clinical Trial Update: Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy [Phase 3]
Phase 3 trial evaluating givinostat in ambulant Duchenne muscular dystrophy completed with posted results.
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CLINICAL_TRIALS trial_result · 2022-12-20Clinical Trial Update: Tamoxifen in Duchenne Muscular Dystrophy [Phase 3]
Phase 3 clinical trial update for Tamoxifen in Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2022-11-18Clinical Trial Update: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 extension study of NS-065/NCNP-01 in boys with Duchenne muscular dystrophy completed; results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-10-19Clinical Trial Update: Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) [Phase 4]
Phase 4 trial update assessing long-term use of viltolarsen in boys with Duchenne muscular dystrophy in clinical practice.