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Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

Lysosomal storage diseases (LSDs) are a group of genetic disorders due to defects in any aspect of lysosomal biology. During the past two decades, different approaches have been introduced for the treatment of these conditions. Among them, enzyme replacement therapy (ERT) represented a major advance...

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Detalles Bibliográficos
Autor principal:	Parenti, Giancarlo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	WILEY-VCH Verlag 2009
Materias:	Bridge the Gap
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3378140/ https://www.ncbi.nlm.nih.gov/pubmed/20049730 http://dx.doi.org/10.1002/emmm.200900036

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