Small molecule therapy for genetic disease / edited by Jess G. Thoene.

"This book summarizes the substantial work that has been accomplished in the treatment of inborn errors of metabolism with simple molecules. These agents are discrete, often of natural origin, and provide predictable therapeutic responses. As such, they avoid many of the practical difficulties...

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Online Access: Full Text (via Cambridge)
Other Authors: Thoene, Jess G.
Format: Electronic eBook
Language:English
Published: Cambridge, UK ; New York : Cambridge University Press, 2010.
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