D-bifunctional Protein Deficiency: 3 Clinical Trials

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2

Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Condition(s):Peroxisome Biogenesis Disorder; Zellweger Spectrum Disorder; RCDP – Rhizomelic Chondrodysplasia Punctata; D-Bifunctional Protein Deficiency; Alpha-Methylacyl-CoA Racemase Deficiency; Peroxisomal Acyl-CoA Oxidase Deficiency; Peroxisomal Acyl-CoA Oxidase 2 Deficiency; ATP Binding Cassette Subfamily D Member 3 Gene Mutation; ACBD5 (AcylCoA Binding Domain 5) Deficiency; Adult Refsum Disease; Sterol Carrier Protein 2 DeficiencyLast Updated:October 31, 2023Recruiting

3

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Condition(s):Mucopolysaccharidosis Disorders; Hurler Syndrome; Hunter Syndrome; Maroteaux Lamy Syndrome; Sly Syndrome; Alpha-Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Glycoprotein Metabolic Disorders; Sphingolipidoses; Recessive Leukodystrophies; Globoid Cell Leukodystrophy; Metachromatic Leukodystrophy; Niemann-Pick B; Niemann-Pick C Subtype 2; Sphingomyelin Deficiency; Peroxisomal Disorders; Adrenoleukodystrophy With Cerebral Involvement; Zellweger Syndrome; Neonatal Adrenoleukodystrophy; Infantile Refsum Disease; Acyl-CoA Oxidase Deficiency; D-Bifunctional Enzyme Deficiency; Multifunctional Enzyme Deficiency; Alpha-methylacyl-CoA Racmase Deficiency; Mitochondrial Neurogastrointestingal Encephalopathy; Severe Osteopetrosis; Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation); Inherited Metabolic DisordersLast Updated:November 3, 2023Recruiting

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