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Clinical studies in lysosomal storage diseases: Past, present, and future

Lysosomal storage disorders (LSDs) consist of over 40 diseases, some of which are amenable to treatment. In this review, we consider the regulatory context in which LSDs studies are performed, highlight design specificities and explore operational challenges. Orphan drug legislations, both in Europe...

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Detalles Bibliográficos
Autor principal: Boudes, Pol F
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Landes Bioscience 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3915565/
https://www.ncbi.nlm.nih.gov/pubmed/25003011
http://dx.doi.org/10.4161/rdis.26690