Yuwu Jiang, Yi Wang, Hui Xiong, Wenhui Li, Rong Luo, Wenxiong Chen, Fei Yin, Junlan Lü, Jianmin Liang, Wan-Jin Chen, Xinguo Lu, Hua Wang, Jihong Tang, Michael Monine, Corinne Makepeace, Xin Jin, Richard Foster, Russell Chin, Zdenek Berger
INTRODUCTION: Spinal muscular atrophy (SMA) is a rare, autosomal recessive, neuromuscular disease that leads to progressive muscular weakness and atrophy. Nusinersen, an antisense oligonucleotide, was approved for SMA in China in February 2019. We report interim results from a post-marketing surveillance phase 4 study, PANDA (NCT04419233), that collects data on the safety, efficacy, and pharmacokinetics of nusinersen in children with SMA in routine clinical practice in China. METHODS: Participants enrolled in PANDA will be observed for 2 years following nusinersen treatment initiation...
May 9, 2024: Advances in Therapy