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ArticleMed (New York, N.Y.)2026

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

Margot A Cousin et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 42735682