Artículo
Novel Therapies for Orphan Diseases
Autor/es | García Fernández, José Manuel
Ortiz Mellet, Carmen |
Departamento | Universidad de Sevilla. Departamento de Química orgánica |
Fecha de publicación | 2019 |
Fecha de depósito | 2022-12-14 |
Publicado en |
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Resumen | "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 ... "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. |
Cita | García Fernández, J.M. y Ortiz Mellet, C. (2019). Novel Therapies for Orphan Diseases. ACS Medicinal Chemistry Letters, 10 (7), 1020-1023. https://doi.org/10.1021/acsmedchemlett.9b00242. |
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