Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant
Joonwoo Rhee,
Jong‐Seol Kang,
Young‐Woo Jo
et al.
Abstract:BackgroundZolgensma is a gene‐replacement therapy that has led to a promising treatment for spinal muscular atrophy (SMA). However, clinical trials of Zolgensma have raised two major concerns: insufficient therapeutic effects and adverse events. In a recent clinical trial, 30% of patients failed to achieve motor milestones despite pre‐symptomatic treatment. In addition, more than 20% of patients showed hepatotoxicity due to excessive virus dosage, even after the administration of an immunosuppressant. Here, we… Show more
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