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

Med 2026 Open access

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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 Donahue, Jany E Dagher
+6 more Jens O. Watzlawik, Wolfdieter Springer, Tejal Parekh, Zachary T. McEachin, Leonard Petrucelli, Bjorn E. Oskarsson
DOI: 10.1016/j.medj.2026.101295
Cited by: 1
References: 33

Fields of Study & Subjects

Amyotrophic Lateral Sclerosis Research Coenzyme Q10 studies and effects RNA Research and Splicing

Publication details

Journal / source
Med
Publisher
Elsevier BV
Volume
7
Issue
10
Pages
101295–101295
Publication date
1 September 2026
Language
EN