Records containing: Muscular Dystrophies
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2020-08-11A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
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CLINICAL_TRIALS study record update · 2020-08-04A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy
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CLINICAL_TRIALS study record update · 2020-07-10A Three-Part FTIH Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Repeat Oral Doses of GSK3439171A, in a Randomized, Double-Blind (Sponsor Unblinded), Placebo-Controlled, Dose Escalation Study and to Evaluate the Effect of Food on a Single Oral Dose of GSK3439171A in Healthy Adult Participants
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CLINICAL_TRIALS study record update · 2020-04-09European Home Mechanical Ventilation Registry
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CLINICAL_TRIALS study record update · 2020-03-18LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2020-03-18A Wearable Device for Breathing Frequency Monitoring: a Pilot Study on Patients With Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2020-03-16Ablelite Pediatric Device Clinical Study
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CLINICAL_TRIALS study record update · 2019-12-10USEFUL: User-centred Assistive SystEm for Arm Functions in neUromuscuLar Subjects
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CLINICAL_TRIALS study record update · 2019-12-09Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy as Diagnostic and Progression Marker Using Multispectral Optoacoustic Tomography
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CLINICAL_TRIALS study record update · 2019-08-19Self-management Training for Parents With Chronic Muscular Dystrophia
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CLINICAL_TRIALS study record update · 2019-08-05Diaphragm Ultrasound in Neuromuscular Disorders
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CLINICAL_TRIALS study record update · 2019-05-17Wearability, Saefty and Usability Assessment for the Upper Limb Exoskeleton BRIDGE/EMPATIA
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CLINICAL_TRIALS study record update · 2019-05-16Prognostic Factors, Morbidity, Mortality and Long Term Survival in Patients With Neuromuscular Disorders Admitted in Intensive Care Unit
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CLINICAL_TRIALS study record update · 2019-01-28A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2018-10-17Psychological Treatment of Disability-Related Pain: Efficacy and Mechanisms
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CLINICAL_TRIALS study record update · 2018-06-14An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS study record update · 2018-05-15Quantification of Muscle Specific microRNAs in the Serum of Patients With Duchenne Muscular Dystrophy (DMD) and Becker (BMD) : Evaluation of the Inters-est of These Biomarkers in Patients Care
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CLINICAL_TRIALS study record update · 2018-04-27Validity and Reliability of Turkish Version of Turkish Version of The Measure of Processes of Care (MPOC 56-20- SP)
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CLINICAL_TRIALS study record update · 2017-10-25Studio Monocentrico in Doppio Cieco Randomizzato Dell'Effetto di Una Miscela di Flavonoidi ed Acidi Grassi Naturali in Pazienti Affetti da Distrofia Muscolare
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CLINICAL_TRIALS study record update · 2017-10-16An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2017-07-18A 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
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CLINICAL_TRIALS study record update · 2017-03-23An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2016-11-07A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration
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CLINICAL_TRIALS study record update · 2016-09-23Development of a New Scale of Quality of Life, Specific of Neuro Muscular Diseases and More Specifically of Steinert Disease, by the Rasch Model Methods.
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CLINICAL_TRIALS study record update · 2015-05-04Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy