Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

202327 citationsJournal Articlebronze Open Access

Authors

Jacqueline Glascock · CURE International UK

Basil T. Darras · Harvard University

Thomas O. Crawford · Johns Hopkins University

Charlotte J. Sumner · Johns Hopkins University

Stephen J. Kolb · The Ohio State University Wexner Medical Center

Christine J. DiDonato · Lurie Children's Hospital

Bakri Elsheikh

Abstract

NF is a promising SMA biomarker with the potential for prognostic, predictive, and pharmacodynamic capabilities. The Working Group has identified needed information to continue efforts toward the validation of NF as a biomarker for SMA.

Topics & Keywords

Neurogenetic and Muscular Disorders Research RNA modifications and cancer Congenital Anomalies and Fetal Surgery

UN Sustainable Development Goals

Partnerships for the goals

Publication Details

Published in: Journal of Neuromuscular Diseases

Volume 10, Issue 5, pp. 937-954

DOI: 10.3233/jnd-230054

Field-Weighted Citation Impact: 7.93