Caption
: Recruiting trial
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; Ongoing trial
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- AUSTRALIA
- New South Wales
- WESTMEAD
- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - AU - (Participation is dependent on local ethics committee approval)
- The Children's Hospital at Westmead
- Institute for Neuroscience and Muscle Research
- More details
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- BELGIUM
- OOST-VLAANDEREN
- GENT
- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - BE - (Participation is dependent on local ethics committee approval)
- Ghent University Hospital - UZGent
- NMRC - K4 - 3e verdieping
- More details
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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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- BELGIUM
- VLAAMS BRABANT
- LEUVEN
- A phase III, randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - BE
- University Hospitals Leuven - Gasthuisberg
- Reference Centre for NeuroMuscular Diseases - UZLeuven
- More details
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- CZECH REPUBLIC
- Capital City Prague
- NOT YET IDENTIFIED - CZ
- A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy -CZ
- Not yet identified - CZ
- More details
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- DENMARK
- Jylland
- NOT YET IDENTIFIED - DK
- A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy - DK
- Not yet identified - DK
- 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
- CHU Paris - Hôpital Necker - Enfants Malades
- Consultation myologie
- 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 Paris Est - Hôpital d'Enfants Armand-Trousseau
- Service de pneumologie pédiatrique
- 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 Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
- Centre de recherche de l'Institut du Cerveau et de la Moelle / Institut de myologie
- More details
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- FRANCE
- ILE-DE-FRANCE
- PARIS
- DELOS : A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients with Duchenne Muscular Dystrophy
- CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
- Centre de recherche de l'Institut du Cerveau et de la Moelle / Institut de myologie
- More details
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- FRANCE
- ILE-DE-FRANCE
- PARIS
- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - FR - (Participation is dependent on local ethics committee approval)
- CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
- Centre de recherche de l'Institut du Cerveau et de la Moelle / Institut de myologie
- More details
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- FRANCE
- POITOU-CHARENTES
- POITIERS
- Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy (MDB-GLN) (Phase II) - Terminated -
- CHU de Poitiers
- Nutrition de l'enfant
- More details
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- FRANCE
- RHONE-ALPES
- NOT YET IDENTIFIED - FR
- A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy - FR
- Not yet identified - FR
- More details
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- FRANCE
- RHONE-ALPES
- NOT YET IDENTIFIED - FR
- DEMAND I: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy - FR Completed
- Not yet identified - FR
- More details
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- GERMANY
- Baden-Württemberg
- FREIBURG
- MD-NET Project R17: Immunosuppressive therapy of the Duchenne Muscular Dystrophy with low dosis Prednisone and Cyclosporin A (Phase III) - Terminated
- Universitätsklinikum Freiburg
- Klinik II - Neuropädiatrie und Muskelerkrankungen
- More details
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- GERMANY
- Baden-Württemberg
- FREIBURG
- DMD114044: A Phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - DE
- Universitätsklinikum Freiburg
- Klinik II - Neuropädiatrie und Muskelerkrankungen
- More details
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- GERMANY
- Baden-Württemberg
- FREIBURG
- PTC124-GD-019-DMD: An open-label phase III study for previously treated Ataluren (PTC124) patients with Nonsense Mutation Dystrophinopathy - DE
- Universitätsklinikum Freiburg
- Klinik II - Neuropädiatrie und Muskelerkrankungen
- More details
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- GERMANY
- Baden-Württemberg
- FREIBURG
- DMD114349: An open-label phase III extension study to evaluate the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - DE
- Universitätsklinikum Freiburg
- Klinik II - Neuropädiatrie und Muskelerkrankungen
- More details
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- MD-NET Projekt P3: Preventive Treatment of left ventricular dysfunction in Duchenne Muscular Dystrophy
- Universitätsklinikum Erlangen
- Kinderkardiologische Abteilung
- More details
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- SUNIMUD: Multicentre, randomized, placebo controlled, double blind phase II/III study to investigate safety and tolerance of Sunphenon Epigallocatechin-Gallate (EGCg) in patients with Duchenne Muscular Dystrophy
- Charité - Universitätsmedizin Berlin (CCM)
- AG Klinische Neuroimmunologie
- More details
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- GERMANY
- Berlin
- NOT YET IDENTIFIED - DE
- FOR-DMD: Double-blind randomized phase III trial to find optimum steroid regimen for Duchenne Muscular Dystrophy - DE
- Not yet identified - DE
- More details
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- GERMANY
- Nordrhein-Westfalen
- ESSEN
- PTC124-GD-007-DMD: A phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy - Terminated
- Universitätsklinikum Essen
- Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ
- More details
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- GERMANY
- Nordrhein-Westfalen
- ESSEN
- DELOS: A Phase III, double-blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of Idebenone in 10-18 year old patients with Duchenne Muscular Dystrophy - DE
- Universitätsklinikum Essen
- Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ
- More details
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- GERMANY
- Nordrhein-Westfalen
- ESSEN
- DMD114117: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne Muscular Dystrophy - DE -
- Universitätsklinikum Essen
- Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ
- More details
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- (FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
- Seconda Università degli Studi di Napoli
- Servizio di Cardiomiologia e Genetica Medica
- 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
- Unità di Malattie Neuromuscolari e Neurodegenerative, Laboratorio di Medicina Molecolare
- More details
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- A Phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - IT
- IRCCS Ospedale Pediatrico Bambino Gesù
- Unità di Malattie Neuromuscolari e Neurodegenerative, Laboratorio di Medicina Molecolare
- More details
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- An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
- IRCCS Ospedale Pediatrico Bambino Gesù
- Unità di Malattie Neuromuscolari e Neurodegenerative, Laboratorio 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.
- Unione Italiana Lotta Distrofia Muscolare
- 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)
- Centro Clinico Nemo
- Unità di Neuropsichiatria Infantile
- More details
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- (FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
- Azienda Ospedaliera Universitaria di Padova - Campus Biomedico Pietro d'Abano
- U.O. di Neuropatologia e Psicopatologia
- More details
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- NETHERLANDS
- Gelderland
- NIJMEGEN
- Een fase II, dubbel blinde, exploratieve, parallel-groep, placebo gecontroleerde klinische studie om de werkzaamheid, veiligheid, verdraagzaamheid en farmacokinetiek te bepalen van twee doseringsschema's van GSK2402968 in ambulante personen met Duchenne spierdystrofie (Deelname is afhankelijk van de goedkeuring van de lokale ethische commissie)
- UMC St Radboud - Oost
- Afdeling Revalidatie
- 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
- Polikliniek 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 PRO044 in Patients With Duchenne Muscular Dystrophy
- LUMC - Leids Universitair Medisch Centrum
- Polikliniek Neurologie
- More details
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- NETHERLANDS
- Zuid-Holland
- LEIDEN
- A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
- LUMC - Leids Universitair Medisch Centrum
- Polikliniek Neurologie
- More details
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- NETHERLANDS
- Zuid-Holland
- LEIDEN
- An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (Phase III)
- LUMC - Leids Universitair Medisch Centrum
- Polikliniek 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 antisense oligoribonucleotide PRO051 in Patients With Duchenne Muscular Dystrophy.
- Prosensa Therapeutics B.V.
- More details
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- POLAND
- Kraków
- NOT YET IDENTIFIED - PL
- A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy - PL
- Not yet identified - PL
- 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 - ES (completed)
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SPAIN
- Cataluña
- ESPLUGUES DE LLOBREGAT
- A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy (terminated)
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SPAIN
- Cataluña
- ESPLUGUES DE LLOBREGAT
- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - ES - (Participation is dependent on local ethics committee approval) (completed)
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SPAIN
- Cataluña
- ESPLUGUES DE LLOBREGAT
- A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy - ES
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SPAIN
- Cataluña
- ESPLUGUES DE LLOBREGAT
- An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - ES
- Hospital Universitari Sant Joan de Déu
- Servicio de Neurología
- More details
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- SPAIN
- Comunidad Valenciana
- VALENCIA
- DELOS : A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients with Duchenne Muscular Dystrophy - ES
- Hospital Universitario y Politécnico La Fe
- Servicio de Neurología (H. La Fe)
- More details
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- DELOS : A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients with Duchenne Muscular Dystrophy - ES
- Hospital Universitario 12 de Octubre
- Sección de Neurología Infantil
- More details
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- SPAIN
- Madrid
- NOT YET IDENTIFIED - ES
- A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy - ES
- Not yet identified - ES
- More details
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- SPAIN
- Madrid
- NOT YET IDENTIFIED - ES
- An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (Phase III) - ES
- Not yet identified - ES
- 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 - SE
- Karolinska University Hospital - Huddinge
- Pediatric Neurology Unit
- More details
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- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - TR - (Participation is dependent on local ethics committee approval)
- Hacettepe university - Faculty of medicine
- Pediatric Nephrology and Rheumatology 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
- Greater London
- LONDON
- Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients (phase I/II)
- UCL Institute of Child Health
- Dubowitz Neuromuscular Centre
- More details
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- UNITED KINGDOM
- Tyne & Wear
- NEWCASTLE UPON TYNE
- A double-blind randomised multi-centre, placebo-controlled trial of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with DMD without echo-detectable left ventricular dysfunction (Phase III)
- Freeman Hospital
- Department of Cardiology
- 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 Genetic Medicine
- More details
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- UNITED KINGDOM
- Tyne & Wear
- NEWCASTLE UPON TYNE
- A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy - UK - (Participation is dependent on local ethics committee approval)
- International Centre for Life
- Institute of Genetic Medicine
- More details
