P.53 Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients

Neuromuscular Disorders - Tập 32 - Trang S62 - 2022
I. Magen1, S. Aharoni2, N. Yacovzada1, I. Tokatly Latzer3, C. Alves4, L. Sagi3, A. Fattal-Valevski3, K. Swoboda4, J. Katz2, E. Bruckheimer2, Y. Nevo2, E. Hornstein1
1Weizmann Institute of Science, Rehovot, Israel
2Schneider Children's Medical Center of Israel, PETACH TIKVA, Israel
3Dana-Dwek Children's Hospital, Tel Aviv, Israel
4Massachusetts General Hospital, Boston, USA