Records containing: Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-08-20The Effect of Intensive Hippotherapy on the Psychomotoric Development of Children With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-08-06Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients
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CLINICAL_TRIALS study record update · 2024-07-23Spinal Muscular Atrophy Patient Registry of the United Kingdom and Ireland
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CLINICAL_TRIALS study record update · 2024-04-18A Multi-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
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CLINICAL_TRIALS study record update · 2024-04-16High-intensity Interval Training in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-04-12A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
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CLINICAL_TRIALS study record update · 2024-04-09Twice Weekly Outpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy Treated With Genetic Based Therapies: A Feasibility Study
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CLINICAL_TRIALS study record update · 2024-03-18Activation of the Spinal Muscular Atrophy Neonatal Screening Program and Integration With Cystic Fibrosis Screening. Feasibility Study
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CLINICAL_TRIALS study record update · 2024-03-08Protocol-LATAM RegistrAME: Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America - a Regional Registry
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CLINICAL_TRIALS study record update · 2024-02-16Study on the Neonatal Screening of Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-02-07Risdiplam Exchange in Patients With Spinal Muscular Atrophy (SMA) Previously and Exclusively Treated With Nusinersen
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CLINICAL_TRIALS study record update · 2024-02-06Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
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CLINICAL_TRIALS study record update · 2024-02-01WREX Outcome Study
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CLINICAL_TRIALS study record update · 2024-01-05A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2023-12-21Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen: NusiMFM
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CLINICAL_TRIALS study record update · 2023-12-21Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
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CLINICAL_TRIALS study record update · 2023-09-13MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-09-07Scientific Basis for a Newborn Screening for Cystinosis and Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-08-31Characterization of the Postural Habits of Wheelchair Users in an Ecological Situation and Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor Integrating an Artificial Intelligence Algorithm
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CLINICAL_TRIALS study record update · 2023-07-07Translation of Motor Function to Daily Activity Performance in Individuals With SMA
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CLINICAL_TRIALS study record update · 2023-05-19Development of a Multidisciplinary Network for Clinical and Laboratory Research for Spinal Muscular Atrophy and Other Rare Motoneuron Diseases
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CLINICAL_TRIALS study record update · 2023-05-10In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-03-22Personalized Medicine for Spinal Muscular Atrophy: a Translational Project to Achieve Patient Specific Therapeutic Guidance.
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CLINICAL_TRIALS study record update · 2023-03-21Clinical Study of Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-03-21Stentrode With Thought-controlled Digital Switch: An Early Feasibility Study of the Safety of an Endovascular Motor Neuroprosthesis in Participants With Severe Upper Limb Impairment.