Description: Rare Disease and Orphan Drugs Journal, with Daniel Scherman as Editor-in-Chief, covers research on genetics, non-hereditary rare diseases, rare cancers, rare tropical diseases, etc. The content of the journal provides in-depth research on ALS, Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidosis, phenylketonuria, thalassemia, osteogenesis imperfect (glass doll), hyperammonemia, organic acids Research on the pathogenesis, drugs, and drug treatments of rare diseases such as hemorrhage and Wils
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