Antisense-based therapy for the treatment of spinal muscular atrophy


One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that… (More)
DOI: 10.1083/jcb.201207087


2 Figures and Tables

Cite this paper

@inproceedings{Rigo2012AntisensebasedTF, title={Antisense-based therapy for the treatment of spinal muscular atrophy}, author={Frank Rigo and Yimin Hua and Adrian R. Krainer and C. F. Bennett}, booktitle={The Journal of cell biology}, year={2012} }