Records containing: Inborn Errors of Metabolism
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CLINICAL_TRIALS study record update · 2020-10-05Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders
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CLINICAL_TRIALS study record update · 2020-03-24Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla
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CLINICAL_TRIALS study record update · 2020-03-16Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute (Dr Schär Medical Nutrition - Mevalia Amino Acids) Mevalia PKU Motion 10 - Tropical or Red Fruits Mevalia PKU Motion 20 - Tropical or Red Fruits
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CLINICAL_TRIALS study record update · 2020-02-17N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia
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CLINICAL_TRIALS study record update · 2018-08-21CD34+Stem Cell Selection for Patients Receiving Partially Matched Family or Matched Unrelated Adult Donor Allogeneic Stem Cell Transplantations for Malignant and Non-Malignant Disease
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CLINICAL_TRIALS study record update · 2017-06-29A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells
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CLINICAL_TRIALS study record update · 2017-06-29Treatment of Early Infantile-Onset Lysosomal Storage Diseases With Fetal Umbilical Cord Blood (UCB) Transplantation
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CLINICAL_TRIALS study record update · 2014-08-20The Early History of Universal Screening for Metabolic Disorders
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CLINICAL_TRIALS study record update · 2014-08-13A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation
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CLINICAL_TRIALS study record update · 2014-07-08A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
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CLINICAL_TRIALS study record update · 2014-05-29Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
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CLINICAL_TRIALS study record update · 2012-01-26Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
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CLINICAL_TRIALS study record update · 2011-02-17Dietary Therapy of Mitochondrial Fatty Acids Oxidation. A Clinical Study of Treatment With Odd Carbons Medium-chain Fatty Acids