Inherited Metabolic Disorders

• CLINICAL_TRIALS research · 2026-03-30
  Clinical 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…

• CLINICAL_TRIALS research · 2026-03-13
  Clinical 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; …

• CLINICAL_TRIALS research · 2026-01-27
  Clinical 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.

• CLINICAL_TRIALS research · 2026-01-13
  Clinical 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…

• CLINICAL_TRIALS research · 2025-12-15
  Clinical 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 · 2025-06-24
  Clinical 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 …

• CLINICAL_TRIALS trial_result · 2024-07-12
  Clinical Trial Update: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases [Phase 2]

  This Phase 2 clinical trial update reports the termination of a haploidentical hematopoietic stem cell transplantation study using reduced-i…

• CLINICAL_TRIALS trial_result · 2021-10-28
  Clinical Trial Update: MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) [Phase 2]

  Phase 2 clinical trial update for MGTA-456 in patients with inherited metabolic disorders undergoing HSCT; study completed with results post…

• CLINICAL_TRIALS trial_result · 2018-10-10
  Clinical Trial Update: A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders [Phase 1]

  Phase 1 clinical trial in pediatric patients with inherited metabolic disorders using a single ProHema-CB product transplant was terminated;…