H. Allen

发表

Results of an Open-Label, Escalating Dose Study To Assess the Safety, Tolerability, and Dose Range Finding of a Single Intrathecal Dose of ISIS-SMNRx in Patients with Spinal Muscular Atrophy (S36.002)

S. Iannaccone, K. Swoboda, D. Vivo, 2013 .