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)
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S. Iannaccone | K. Swoboda | D. Vivo | J. Montes | D. Norris | C. Chiriboga | K. Bishop | B. Darras | K. Alexander | F. Bennett | H. Allen | R. Parad | S. Johnson