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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 14
Selected: 0
NCT IDTitle
NCT02999984Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
NCT06149403A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
NCT03047369The Myelin Disorders Biorepository Project
NCT05687474Baby Detect : Genomic Newborn Screening
NCT01560182Gene Therapy for Metachromatic Leukodystrophy (MLD)
NCT05368038ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05354219Validation and Reliability of Iris Cameras in Mucopolysaccharidoses
NCT01852071Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
NCT03392987A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
NCT02453477Gene Therapy for Transfusion Dependent Beta-thalassemia
NCT03765632Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
NCT04283227OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
NCT01380990Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
NCT04201405Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA