Impact of Oligonucleotides - 2: Expert Says Spinraza Changed SMA Treatment Paradigm, Has High Expectation for Zolgensma Too

Kayoko Saito, Director, Institute of Clinical Genomics, Tokyo Women’s Medical University “Without an effective treatment, 95% of children with spinal muscular atrophy (SMA) require a tracheotomy by their second birthday, but babies who received Spinraza (nusinersen) in clinical trials were...read more