Records containing: Niemann-Pick Disease
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 · 2023-04-12Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2023-02-13Biomarker for Niemann Pick Type C Disease (NPC1/NPC2) an International, Multicenter, Epidemiological Study
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CLINICAL_TRIALS study record update · 2022-10-25A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
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CLINICAL_TRIALS study record update · 2017-04-04ClinicalTrials.gov study NCT02120235
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CLINICAL_TRIALS study record update · 2016-06-23Phase I/II Trial Of Hematopoietic Stem Cell Transplant (HSCT) For Children With A Genetic Disease Of Blood Cells Without An HLA-Matched Sibling Donor
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CLINICAL_TRIALS study record update · 2015-03-19A Phase I, Single-Center, Single Dose, Dose Escalation Study of Recombinant Human Acid Sphingomyelinase (rhASM) in Adults With Acid Sphingomyelinase Deficiency (ASMD)
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CLINICAL_TRIALS study record update · 2011-06-10Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation