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"title": "A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene",
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"title": "A Phase 2 Open-label Study to Evaluate Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Primary or Secondary Myelofibrosis",
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"title": "A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy",
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"title": "The Biosonographic Index - A Novel Index for the Early Detection of Acute Kidney Injury After Vascular Surgery - an Exploratory Study.",
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