Orphan Drugs
Author:
Publisher
Springer International Publishing
Link
http://link.springer.com/content/pdf/10.1007/978-3-319-05392-9_137
Reference83 articles.
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3. Bali RK, Bos L, Gibbons MC, Ibell SR (2013) Rare diseases in the age of health 2.0. Springer, Heidelberg
4. Bellettato CM, Scarpa M (2010) Pathophysiology of neuropathic lysosomal storage disorders. J Inherit Metab Dis 33(4):347–362
5. Biegstraaten M, van Schaik IN, Aerts JM, Hollak CE (2008) ‘Non-neuronopathic’ Gaucher disease reconsidered. Prevalence of neurological manifestations in a Dutch cohort of type I Gaucher disease patients and a systematic review of the literature. J Inherit Metab Dis 31(3):337–349
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