Novel Therapies for Orphan Diseases

descriptionPublicationkeyboard_double_arrow_right Article , Other literature type 18 Jun 2019 Spain English Publisher:American Chemical Society (ACS)Journal:ACS Medicinal Chemistry Letters, volume 10, pages 1,020-1,023 (issn: 1948-5875, eissn: 1948-5875,

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Authors: José M. García Fernández; Carmen Ortiz Mellet;

doi: 10.1021/acsmedchemlett.9b00242

pmid: 31312401

pmc: PMC6627715

handle: 10261/204092 , 11441/140467

Novel Therapies for Orphan Diseases

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Abstract

"Orphan" does not mean infrequent: over 7000 rare diseases affect millions of individuals. The US Orphan Drug Act and analogous regulations have succeeded at accelerating the development of novel therapies, but high prices threaten sustainability. Lysosomal storage disorders serve here to illustrate the light and shadows of this burgeoning field.

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