Inherited Metabolic Disorders
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: Caregiving Networks Across Disease Context and the Life Course
The National Human Genome Research Institute sponsors a study examining caregiving networks across disease contexts and life stages, current…
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders [Phase 2]
Phase 2 clinical trial evaluating cord blood transplant in children and young adults with blood cancers and select non-malignant disorders; …
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CLINICAL_TRIALS research · 2026-01-27Clinical Trial Update: AI for Newborn Metabolic Screening [NA]
Clinical trial update on AI-assisted newborn metabolic screening for inherited metabolic disorders; the study status is not yet recruiting.
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CLINICAL_TRIALS research · 2026-01-13Clinical Trial Update: Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Clinical trial update describing a data collection study of patients with non-malignant disorders undergoing umbilical cord blood transplant…
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CLINICAL_TRIALS research · 2025-12-15Clinical Trial Update: Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT [Phase 2]
Phase 2 study evaluating reduced-intensity conditioning for non-malignant disorders undergoing umbilical cord blood transplantation (UCBT), …
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CLINICAL_TRIALS research · 2025-06-24Clinical Trial Update: TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children [Phase 2]
Phase 2 clinical trial evaluating TCR alpha/beta T-cell-depleted haploidentical hematopoietic cell transplantation in children with primary …