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Molecular Genetics and Metabolism, ISSN 1096-7192, 03/2016, Volume 117, Issue 3, pp. 313 - 321
Journal Article
Journal of Human Genetics, ISSN 1434-5161, 2018, Volume 64, Issue 2, pp. 139 - 143
Gene therapies for lysosomal storage diseases (LSD) and peroxisomal diseases (PD) are rapidly advancing. Most LSDs and PDs are characterized by brain... 
PHASE | GENETICS & HEREDITY | CHILDREN | VECTOR | Enzyme Replacement Therapy | Genetic Therapy | Animals | Lysosomal Storage Diseases - therapy | Peroxisomal Disorders - genetics | Humans | Peroxisomal Disorders - therapy | Lysosomal Storage Diseases - genetics
Journal Article
Journal Article
Neurobiology of Aging, ISSN 0197-4580, 2015, Volume 36, Issue 3, pp. 1451 - 1461
Journal Article
Current Treatment Options in Oncology, ISSN 1527-2729, 12/2014, Volume 15, Issue 4, pp. 539 - 550
Journal Article
Case Reports in Gastroenterology, ISSN 1662-0631, 06/2018, Volume 12, Issue 2, pp. 360 - 372
Zellweger spectrum disorders (ZSDs), a subgroup of peroxisomal biogenesis disorders, have a generalized defect in peroxisome function. Liver disease in ZSDs... 
Case Series | Bile acid synthesis | Peroxisomal biogenesis disorder | Liver function | Zellweger spectrum disorder | Cholic acid | Long-term treatment | Urine | Enzymes | Liver diseases | Laboratories | Biosynthesis | FDA approval | Patients | Fatty acids | Hospitals | Metabolites | Biopsy | Age | Bile
Journal Article
Developmental Disabilities Research Reviews, ISSN 1940-5510, 06/2013, Volume 17, Issue 3, pp. 187 - 196
Journal Article
Journal Article