COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY
摘要
Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
申请公布号
EP2872493(A4)
申请公布日期
2016.04.13
申请号
EP20130817021
申请日期
2013.07.12
申请人
INDIANA UNIVERSITY RESEARCH AND TECHNOLOGY CORPORATION;THE BRIGHAM AND WOMEN'S HOSPITAL, INC.;THE UNIVERSITY OF MASSACHUSETTS
发明人
ANDROPHY, ELLIOT J.;CHERRY, JONATHON J.;CUNY, GREGORY D.;GLICKSMAN, MARCIE A.
