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Published on May 13, 2013
This presentation was originally given at the 2008 Pitlochry Scotland Pachyonychia Congenita Patient Support Meeting and depicts the first in human, first in skin siRNA TD101 clinical trial in the patient's point of view. Learn more at www.pachyonychia.org.
Leachman SA et al., (2010) First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder. Molecular Therapy 8:442-446 (PubMed ID: 19935778)