Pediatric Endocrinology Reviews
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Volume 17 Sup 1 - shop
Volume 17.2 - shop
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Lysosomal Storage Disorders: old diseases, present
Orphan Drug Development Gregory M. Pastores, MD; P
Clinical Studies in Lysosomal Storage Diseases: Pa
Oral Small Molecule Therapy for Lysosomal Storage
Hematopoietic Stem Cell Transplant for Lysosomal S
Gene Therapy for Lysosomal Storage Disorders Nelso
Legal and Regulatory Aspects of Orphan Drugs Segev
Orphan Drug Development and the Impact on Non-Medi
