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Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

Orphan drugs are often approved under exceptional circumstances, requiring submission of additional data on safety and effectiveness through registries. These registries are mainly focused on one drug only and data is frequently incomplete. Some registries also address phenotypic heterogeneity and n...

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Detalles Bibliográficos
Autores principales:	Hollak, Carla EM, Aerts, Johannes MFG, Aymé, Ségolène, Manuel, Jeremy
Formato:	Texto
Lenguaje:	English
Publicado:	BioMed Central 2011
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3102605/ https://www.ncbi.nlm.nih.gov/pubmed/21496291 http://dx.doi.org/10.1186/1750-1172-6-16

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