HIGHLIGHTS
- who: BMC Neuroscience and collaborators from the Address: Department of Veterinary Pathobiology, Bond Life Sciences Center, University of Missouri, Columbia MO, USA and Department of have published the article: Bio Med Central, in the Journal: (JOURNAL)
- what: The authors demonstrate that subcutaneous-administration of a read-through inducing compound (TC007) to an intermediate SMA model (Smn-/-; SMN2+/+; SMNĪ7) had beneficial effects on muscle fiber size and gross motor function. The authors demonstrate that subcutaneous administration of TC007, while not extending survival, results in improved molecular and cellular aspects of the SMA phenotype, as well . . .
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