%0 Journal Article %A Ekins, Sean %A Wood, Jill %A sames, Lori %D 2013 %T Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery %U https://figshare.com/articles/journal_contribution/Multifaceted_roles_of_ultra_rare_and_rare_disease_patients_parents_in_drug_discovery/782257 %R 10.6084/m9.figshare.782257.v1 %2 https://ndownloader.figshare.com/files/1182471 %K sanfilippo syndrome %K rare diseases %K charcot-marie-tooth %K giant axonal neuropathy %K patient advocate %K parent advocate %K Medicine %K Neuroscience %K Pharmacology %X

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PMID:
23968993
Drug Discov Today. 2013 Aug 19. pii: S1359-6446(13)00269-9. doi: 10.1016/j.drudis.2013.08.006. [Epub ahead of print]
Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery.
Wood J, Sames L, Moore A, Ekins S.

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