Records containing: Spinal Muscular Atrophy Type I
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 · 2026-03-02A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1
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CLINICAL_TRIALS study record update · 2025-10-06Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
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CLINICAL_TRIALS study record update · 2025-04-25A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
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CLINICAL_TRIALS study record update · 2025-03-07Impact of Motor and Oral Motor Function on Quality of Life in Children With Type I Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-10-09Therapeutic Management and Current Situation Related to the Use of Resources and Costs of Spinal Muscular Atrophy (SMA) in Spain: A Retrospective, Multicenter Analysis Based on Medical Record Review. TREaSURE Study
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CLINICAL_TRIALS study record update · 2024-09-19A Multicenter, Nonrandomized, Open-label,Dose Escalation Clinical Trial to Assess the Safety and Efficacy of EXG001 307 After Intravenous Injection in Patients with Spinal Muscular Atrophy Type 1
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CLINICAL_TRIALS study record update · 2024-09-19The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-07-22Exploring the Feasibility and Utility of In-home Body Weight Support Harness System Use in Children Treated for Spinal Muscular Atrophy: a Pilot Study
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CLINICAL_TRIALS study record update · 2022-01-11Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
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CLINICAL_TRIALS study record update · 2015-06-15Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)
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CLINICAL_TRIALS study record update · 2010-07-20Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular Atrophy