Ontology highlight
ABSTRACT:
SUBMITTER: Chaytow H
PROVIDER: S-EPMC8324491 | biostudies-literature | 2021 Jul
REPOSITORIES: biostudies-literature
Chaytow Helena H Faller Kiterie M E KME Huang Yu-Ting YT Gillingwater Thomas H TH
Cell reports. Medicine 20210721 7
Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the most severe cases and when left untreated, leads to death within the first two years of life. Recent therapeutic advances have given hope to families and patients by compensating for the deficiency in survival motor neuron (SMN) protein via gene therapy or other genetic manipulation. However, it is now apparent that none of these therapies will cure SMA alone. In this review, we discuss the three currently ...[more]