专利名称：COMBINATION THERAPIES FOR
TREATMENT OF SPINAL MUSCULARATROPHY
发明人：Elliot J. Androphy,Kevin Hodgetts,Alyssa
Nicole Calder
申请号：US15440727申请日：20170223
公开号：US20170239225A1公开日：20170824
摘要：Disclosed herein are compositions and methods for treatment of spinalmuscular atrophy (SMA). In certain embodiments, compounds are provided that increasefull-length survival of motor neuron (SMN) protein production by an SMN2 gene.
申请人：Indiana University Research and Technology Corporation,The Brigham andWomen's Hospital, Inc.
地址：Indianapolis IN US,Boston MA US
国籍：US,US
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