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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: 16
Selected: 0
NCT IDTitle
NCT04105166Gene Therapy for Pyruvate Kinase Deficiency (PKD)
NCT03766386The Natural History of Danon Disease
NCT03812263A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
NCT04248439Gene Therapy for Fanconi Anemia, Complementation Group A
NCT06092034A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
NCT05885412A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
NCT05548855Natural History of Danon Disease
NCT03814408A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
NCT06282432Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
NCT03882437Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
NCT04525352A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
NCT06214507Danon Disease Natural History Study
NCT04069533Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
NCT06422351Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
NCT04437771Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
NCT03825783A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I