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Clinical Trials for Peroxisomal Disorders

• Hydroxychloroquine Administration for Reduction of Pexophagy
• MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
• Betaine and Peroxisome Biogenesis Disorders
• Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
• Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
• Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
• Study of Bile Acids in Patients With Peroxisomal Disorders