Review
Open access
Oct 2026
An AI-Enabled Translational Drug Discovery Framework for NALCN Channelopathy: From High-Throughput Screening to Therapeutic Candidate
Background More than 90% of rare diseases lack an approved therapy, partly reflecting economic barriers in developing treatments for small patient populations. A common bottleneck is costly and resource-intensive hit-to-lead prioritization. Congenital contractures of the limbs and face, hypotonia, and developmental del...
N. Thongsepee, Jeremy A. Tanner, Srikanth R. Polusani et al.
· bioRxiv · 0 citations