N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy. (linkinghub.elsevier.com)

Authors: Cousin MA … Oskarsson B Journal: Cell Med Published: 2026-09-14 DOI: https://doi.org/10.1016/j.medj.2026.101295