Authors
Kaczmarek A., Schneider S., Wirth B., Riessland M.
Journal
Expert Opinion on Investigational Drugs, volume 24, issue 7, pages 867-881
Publication date
July 2015
Abstract
Currently, there is no cure available for the hereditary neurodegenerative disease proximal spinal muscular atrophy (SMA), which is the number one genetic killer in early childhood. However, growing knowledge of SMA pathophysiology has opened new avenues for potential therapeutic interventions.
DOI link
10.1517/13543784.2015.1038341