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