Records containing: Sphingolipidoses
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-01-07MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
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CLINICAL_TRIALS study record update · 2025-10-24Sphingosine Phosphate Lyase Insufficiency Syndrome - Observational Study and Patient Registry (International)
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CLINICAL_TRIALS study record update · 2021-05-28Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease: An International, Multicenter, Epidemiological Protocol
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CLINICAL_TRIALS study record update · 2021-03-17Natural History Study of Children With Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2017-05-12Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2011-07-13Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation