Work

N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy

Year: 2026

Type: article

Source: Med

DOI: 10.1016/j.medj.2026.101295

Authors: Margot A. Cousin, Aditi I. Dagli, Laurence Mignon, Jaimin S. Shah, Mercedes Prudencio, Tania F. Gendron, Angita Jain, Evan Udine, Marka van Blitterswijk, Erik H. Middlebrooks, Megan H. Donahue, Jany E. Dagher, Jens O. Watzlawik, Wolfdieter Springer, Tejal Parekh, Zachary T. McEachin, Leonard Petrucelli, Björn Oskarsson

Institutions: Mayo Clinic, Orlando Health, Mayo Clinic in Florida, University of Central Florida, Emory University, University of Miami

Language: English

Topic: Amyotrophic Lateral Sclerosis Research

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: Georgia Clinical and Translational Science Alliance, National Institutes of Health, National Institute of Neurological Disorders and Stroke, National Center for Advancing Translational Sciences

Awards: Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS, G3667020645, G7894441833, G9000356365, Institutional Career Development Core

OpenAlex ID: W7212601585

FWCI: 4.197

Cites: 33

Cited by: 1

Related to: 10