Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study

Crawford, T. O., Day, J. W., De Vivo, D. C., Krueger, J. M., Mercuri, E., Nascimento, A., Pasternak, A., Mazzone, E. S., Duong, T., Song, G., Marantz, J. L., Baver, S., Yu, D., Liu, L., & Darras, B. T. (2024). Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study. Frontiers in Neurology, 15. https://doi.org/10.3389/fneur.2024.1419791

Authors:

Thomas O. Crawford

John Day

Darryl C. De Vivo

Jena M. Krueger

Eugenio Mercuri

A. Nascimento

Amy Pasternak

Elena Mazzone

Tina Duong

Guochen Song

Jing L. Marantz