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Pediatric Endocrinology Reviews : PER|January 2, 2014
Lysosomal storage disorders: old diseases, present and future challengesAndrés D Klein, Anthony H FutermanPediatric Endocrinology Reviews : PER|January 2, 2014
Orphan drug developmentGregory M Pastores, Punita GuptaPediatric Endocrinology Reviews : PER|January 2, 2014
Oral small molecule therapy for lysosomal storage diseasesNeal J WeinrebPediatric Endocrinology Reviews : PER|January 2, 2014
Legal and regulatory aspects of orphan drugsSegev Shani, Zohar YahalomPediatric Endocrinology Reviews : PER|January 2, 2014
Orphan drug development and the impact on non-medical support groupsJeremy Manuel, Tanya Collin-HistedPediatric Endocrinology Reviews : PER|July 13, 2012
Brain development is a multi-level regulated process--the case of the OTX2 geneGalia Gat-YablonskiPediatric Endocrinology Reviews : PER|July 13, 2012
Sport training and the growth and pubertal maturation of young athletesRobert M Matina, Alan D RogolPediatric Endocrinology Reviews : PER|February 2, 2010
Autoimmune polyendocrinopathy-candidosis-ectodermal dystrophy (APECED)--a diagnostic and therapeutic challengeJ Jääskeläinen, J PerheentupaPageof 77