Orphanet Journal of Rare Diseases
The official journal of Orphanet, the international portal for rare diseases and orphan drugs, Orphanet Journal of Rare Diseases is an open access, online journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high quality reviews solicited from experts in the field and also considers research on rare diseases and results of clinical trials of exceptional interest.
ReviewTopical Collection Lysosomal storage diseases
ReviewTopical Collection Pediatric neuromuscular diseases
ResearchTopical Collection Inherited metabolic diseases
To view the rest of this content please follow the download PDF link above.