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Transposon's TPN-101 selected for testing in HEALEY ALS trial

Summary by ALS News Today
Oral small molecule TPN-101 has been selected for testing in amyotrophic lateral sclerosis (ALS) as part of the HEALEY ALS platform trial. The decision to include TPN-101 in the platform trial (NCT04297683) was based on the treatment’s unique mechanism of action and final data from a Phase 2 clinical trial (NCT04993755) in adults with ALS or frontotemporal dementia (FTD) related to C9orf72 mutations, developer Transposon Therapeutics said. The c…
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ALS News Today broke the news in on Monday, June 2, 2025.
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