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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...

Cur síos iomlán

Na minha lista:
Sonraí Bibleagrafaíochta
Príomhúdar: Boudes, Pol F
Formáid: Artigo
Teanga:Inglês
Foilsithe: Landes Bioscience 2013
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3915565/
https://ncbi.nlm.nih.gov/pubmed/25003011
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/rdis.26690
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