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Recruiting trial
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National clinical trial(s)
FRANCE
AUVERGNE-RHONE-ALPES
NOT YET IDENTIFIED - FR
FRANCE
AUVERGNE-RHONE-ALPES
NOT YET IDENTIFIED - FR
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy-FR
NOT YET IDENTIFIED - FR
GERMANY
Baden-Württemberg
FREIBURG
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 - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
GERMANY
Baden-Württemberg
FREIBURG
An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
GERMANY
Baden-Württemberg
FREIBURG
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy - DE - Completed
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
GERMANY
Baden-Württemberg
FREIBURG
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
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 - DE
Universitätsklinikum Essen
Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ
ITALY
LAZIO
ROMA
An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - IT
IRCCS Ospedale Pediatrico Bambino Gesù
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare
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
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
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
SPAIN
Cataluña
ESPLUGUES DE LLOBREGAT
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy - ES (Completed)
Hospital Universitari Sant Joan de Déu
Servicio de neurología
SPAIN
Comunidad Valenciana
VALENCIA
A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - ES
Hospital Universitario y Politécnico La Fe
Servicio de Neurología
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
A Phase III Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - UK
Newcastle upon Tyne Hospitals NHS Trust
Institute of Genetic Medicine
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy (phase III) - UK
Newcastle upon Tyne Hospitals NHS Trust
Institute of Genetic Medicine
UNITED STATES
New Jersey
SOUTH PLAINFIELD
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy (Phase III) - FR
PTC Therapeutics Inc.
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
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
GERMANY
Baden-Württemberg
FREIBURG
MD-NET Project R17: Immunosuppressive therapy of the Duchenne Muscular Dystrophy with low dosis Prednisone and Cyclosporin A (Phase III) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
ITALY
LAZIO
ROMA
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
ITALY
LAZIO
ROMA
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
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
Astrid Lindgren Children's Hospital - Karolinska
Pediatric Neurology
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
PTC124-GD-007-DMD: A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - UK
Newcastle upon Tyne Hospitals NHS Trust
Institute of Genetic Medicine
FRANCE
ILE-DE-FRANCE
GARCHES
Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease - FR
Raymond Poincaré Hospital
FRANCE
ILE-DE-FRANCE
GARCHES
Establishment of a Non-invasive and Indirect Measure of Volitional Pimax (PI MAX STIM) - FR
Raymond Poincaré Hospital
FRANCE
ILE-DE-FRANCE
GARCHES
Protocole de Validation Clinique du Logiciel d'Interface Cerveau-Ordinateur RoBIK Pour la Communication Des Patients Atteints de Maladie Neuromusculaires (MNM) - FR
Raymond Poincaré Hospital
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
Multinational clinical trial(s)
UNITED STATES
New Jersey
SOUTH PLAINFIELD
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy - coordination
PTC Therapeutics Inc.
UNITED STATES
New Jersey
SOUTH PLAINFIELD
A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy (coordination)
PTC Therapeutics Inc.
UNITED STATES
New Jersey
SOUTH PLAINFIELD
A phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (coordination)
PTC Therapeutics Inc.
FRANCE
ILE-DE-FRANCE
PARIS