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Recent reporting, analysis and explainers from the ALS desk.

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First mutation-targeted RNA therapy improves rare ALS case

A man with slowly progressing ALS caused by a rare CHCHD10 mutation showed improved motor-function scores and a biomarker associated with nerve damage returned to the normal reference range one year after receiving a mutation-targeted antisense oligonucleotide treatment.

2026-09-21T03:03:44+05:30
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ALS Mouse Study Supports Restoring Multiple TDP-43 Targets

A bioRxiv preprint reports that restoring several TDP-43 cryptic targets, including Unc13a, protected spinal motor neurons in a mouse model of early-stage ALS more effectively than targeting Unc13a alone.

2026-09-19T00:17:26+05:30