480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes
Open Access
- 1 May 2016
- journal article
- Published by Elsevier BV in Molecular Therapy
- Vol. 24, S190
- https://doi.org/10.1016/s1525-0016(16)33289-0