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Clinical Trials in Rare Disease: Challenges and Opportunities

The neuronal ceroid lipofuscinoses constitute one of many groups of rare childhood diseases for which disease-modifying treatments are non-existent. Disease-specific barriers to therapeutic success include incomplete understanding of disease pathophysiology and limitations of treatments that cannot...

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Bibliografiske detaljer
Main Authors: Augustine, Erika F., Adams, Heather R., Mink, Jonathan W.
Format: Artigo
Sprog:Inglês
Udgivet: 2013
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3964003/
https://ncbi.nlm.nih.gov/pubmed/24014509
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073813495959
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