Intraperitoneal delivery of a novel drug-like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy

Osman, EY; Rietz, A; Kline, RA; Cherry, JJ; Hodgetts, KJ; Lorson, CL; Androphy, EJ

Lorson, CL (reprint author), Univ Missouri, Dept Vet Pathobiol, Coll Vet Med, Bond Life Sci Ctr, Columbia, MO 65201 USA.; Androphy, EJ (reprint author), Indiana Univ Sch Med, Dept Dermatol, Indianapolis, IN 46202 USA.

SCIENTIFIC REPORTS, 2019; 9 ():

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder that causes progressive muscle weakness and is the leading genetic ......

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