Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant

• Main Authors: Joonwoo Rhee (Author), Jong‐Seol Kang (Author), Young‐Woo Jo (Author), Kyusang Yoo (Author), Ye Lynne Kim (Author), Sang‐Hyeon Hann (Author), Yea‐Eun Kim (Author), Hyun Kim (Author), Ji‐Hoon Kim (Author), Young‐Yun Kong (Author)
• Format: Book
• Published: Wiley, 2024-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Zolgensma 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...