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Invention E-109-2010/0

Arylthiazolyl Piperidines and Related Compounds as Modulators of Survival Motor
Neuron (SMN) Protein Production

Lead Inventor: Juan Marugan (NCATS)
Inventors: Elliotn Adrophy (University of Massachusetts Medical School), Jonathan Cherry (University of Massachusetts Medical School), Noel Southall (NCATS) Steven Titus (NCATS), Jingbo Xiao (NCATS), Wei Zheng (NCATS)
Ref. No.: E-109-2010/0

Abstract: This technology discloses compounds that modulate the amount of survival motor neuron protein (SMN). Low levels of SMN protein are associated with spinal muscular atrophy (SMA), which constitutes a group of inherited diseases that cause progressive muscle degeneration leading to death. Consequently, therapeutic inventions have focused on increasing SMN protein levels. This invention discloses novel arylthiazolyl piperidines, which are shown to be modulators of SMN production. This invention also discloses methods of treating SMA by administering SMN protein modulators.


Discovery, Synthesis, and Biological Evaluation of Novel SMN Protein ModulatorsJournal of Medicinal Chemistry • Sept. 22, 2011 • Probe Development Branch, NCATS Chemical Genomics Center

Last updated: 07-21-2016
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