Caption
: Recruiting trial
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; Ongoing trial
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- BELGIUM
- VLAAMS BRABANT
- LEUVEN
- Phase IIa double-blind randomized placebo-controlled single-centre study to assess the efficacy and tolerability of SNT-MC17/idebenone in 8-16 year-old males with cardiac dysfunction associated with DMD -Completed-
- University Hospitals Leuven - Gasthuisberg
- Pediatric Neurology
- More details
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- BELGIUM
- VLAAMS BRABANT
- LEUVEN
- A Phase II Open-Label Extension Study to Obtain Long-Term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001 (DELPHI Extension).
- University Hospitals Leuven - Gasthuisberg
- Pediatric Neurology
- More details
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- BELGIUM
- VLAAMS BRABANT
- LEUVEN
- A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy.
- University Hospitals Leuven - Gasthuisberg
- Reference Centre for NeuroMuscular Diseases - UZLeuven
- More details
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- BELGIUM
- VLAAMS BRABANT
- LEUVEN
- A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy.
- University Hospitals Leuven - Gasthuisberg
- Reference Centre for NeuroMuscular Diseases - UZLeuven
- More details
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- FRANCE
- ILE DE FRANCE
- PARIS
- A Phase IIb Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- CHU Hôpital Pitié-Salpêtrière
- Unité mixte de recherche en santé 974 / Université Pierre et Marie Curie
- More details
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- FRANCE
- ILE DE FRANCE
- PARIS
- Nemucough : Evaluation of a Mechanical Insufflation-Exsufflation Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders: a Prospective, Randomized, Controlled, Multicenter Study (Phase III)
- CHU Hôpital d'Enfants Armand-Trousseau
- Service de pneumologie pédiatrique
- More details
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- FRANCE
- ILE DE FRANCE
- PARIS
- Evaluation of a Mechanical Insufflation-exsufflation Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders: a Prospective, Randomized, Controlled, Multicenter Study (Phase III)
- CHU Hôpital d'Enfants Armand-Trousseau
- Service de pneumologie pédiatrique
- More details
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- FRANCE
- ILE DE FRANCE
- PARIS
- Assessment of the effect of perindopril orodispersible tablet at the dose of 0.150 mg/kg/day on muscular and myocardic functions in the early stage of Duchenne Muscular Dystrophy: a two-year, double-blind, randomised, placebo-controlled study
- Hopital Necker - Enfants Malades
- Consultation myologie
- More details
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- GERMANY
- Baden-Württemberg
- FREIBURG
- A phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- Universitätsklinikum Freiburg
- Klinik II - Neuropädiatrie und Muskelerkrankungen
- More details
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- GERMANY
- Nordrhein-Westfalen
- ESSEN
- A phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- Universitätsklinikum Essen
- Pädiatrische Neurologie
- More details
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- A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- IRCCS Ospedale Pediatrico Bambino Gesù
- U.O. di Medicina Molecolare
- More details
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- Effects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study.
- Centro Malattie Neuromuscolari
- More details
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- A double-blinded randomized placebo controlled study of daily Pentoxifylline as a rescue treatment in Duchenne muscular dystrophy (CLOSED)
- Fondazione Istituto Neurologico "C. Mondino" - IRCCS
- Centro di Riferimento Regionale per le Malattie Neuromuscolari dell'Età Evolutiva
- More details
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- NETHERLANDS
- Zuid-Holland
- LEIDEN
- Exploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.
- LUMC - Leids Universitair Medisch Centrum
- Afdeling Neurologie
- More details
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- NETHERLANDS
- Zuid-Holland
- LEIDEN
- A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of PRO051 in Patients With Duchenne Muscular Dystrophy
- Prosensa B.V.
- More details
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- NETHERLANDS
- Zuid-Holland
- LEIDEN
- A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of antisense oligoribonucleotide PRO051 in Patients With Duchenne Muscular Dystrophy.
- Prosensa B.V.
- More details
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- SPAIN
- Cataluña
- ESPLUGUES DE LLOBREGAT
- A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SWEDEN
- Stockholms läns landsting
- STOCKHOLM
- A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy
- Karolinska University Hospital
- Pediatric Neurology Unit
- More details
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- UNITED KINGDOM
- Greater London
- LONDON
- Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 (morpholino oligomer directed against exon 51)
- Hammersmith Hospital
- Dubowitz Neuromuscular Unit (Imperial College London)
- More details
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- UNITED KINGDOM
- Tyne & Wear
- NEWCASTLE UPON TYNE
- A phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- International Centre for Life
- Institute of Human Genetics
- More details
