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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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| Main Authors: | , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
BioMed Central
2011
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| Teme: | |
| Online dostop: | 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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