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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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Bibliografiske detaljer
Main Authors: Hollak, Carla EM, Aerts, Johannes MFG, Aymé, Ségolène, Manuel, Jeremy
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2011
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3102605/
https://ncbi.nlm.nih.gov/pubmed/21496291
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-6-16
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