The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Singh, RN; Ottesen, EW; Singh, NN

Singh, RN; Singh, NN (corresponding author), Iowa State Univ, Dept Biomed Sci, 1800 Christensen Dr, Ames, IA 50011 USA.

NEUROSCIENCE INSIGHTS, 2020; 15 ():

Abstract

Spinal muscular atrophy (SMA) is 1 of the leading causes of infant mortality. SMA is mostly caused by low levels of Survival Motor Neuron (SMN) protei......

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