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

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

Open source

DOI
10.1016/j.medj.2026.101295
Published
2026-09
Container
Med
Publisher
Elsevier BV
Open access
unknown

Credibility signals

uncertain Score 64/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

Cite this work

BibTeX

@article{allodium:10.1016/j.medj.2026.101295,
  title = {N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy},
  author = {Margot A. Cousin and Aditi I. Dagli and Laurence Mignon and Jaimin S. Shah and Mercedes Prudencio and Tania F. Gendron and Angita Jain and Evan Udine and Marka van Blitterswijk and Erik H. Middlebrooks and Megan H. Donahue and Jany E. Dagher and Jens O. Watzlawik and Wolfdieter Springer and Tejal Parekh and Zachary T. McEachin and Leonard Petrucelli and Björn Oskarsson},
  year = {2026},
  journal = {Med},
  doi = {10.1016/j.medj.2026.101295},
  url = {https://doi.org/10.1016/j.medj.2026.101295}
}

RIS

TY  - JOUR
TI  - N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy
AU  - Margot A. Cousin
AU  - Aditi I. Dagli
AU  - Laurence Mignon
AU  - Jaimin S. Shah
AU  - Mercedes Prudencio
AU  - Tania F. Gendron
AU  - Angita Jain
AU  - Evan Udine
AU  - Marka van Blitterswijk
AU  - Erik H. Middlebrooks
AU  - Megan H. Donahue
AU  - Jany E. Dagher
AU  - Jens O. Watzlawik
AU  - Wolfdieter Springer
AU  - Tejal Parekh
AU  - Zachary T. McEachin
AU  - Leonard Petrucelli
AU  - Björn Oskarsson
PY  - 2026
JO  - Med
DO  - 10.1016/j.medj.2026.101295
UR  - https://doi.org/10.1016/j.medj.2026.101295
ER  - 

APA

Cousin, M. A., Dagli, A. I., Mignon, L., Shah, J. S., Prudencio, M., Gendron, T. F., Jain, A., Udine, E., Blitterswijk, M. V., Middlebrooks, E. H., Donahue, M. H., Dagher, J. E., Watzlawik, J. O., Springer, W., Parekh, T., McEachin, Z. T., Petrucelli, L., & Oskarsson, B. (2026). N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy. Med. https://doi.org/10.1016/j.medj.2026.101295

Source records