Ionis Pharmaceuticals, Inc.
Clinical trials sponsored by Ionis Pharmaceuticals, Inc., explained in plain language.
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Down syndrome Alzheimer's drug study halted after just one volunteer
Disease control TerminatedThis study tested a new drug called ION269 in adults with Down syndrome who also had signs of Alzheimer's-related brain changes. The main goal was to check if the drug was safe and tolerable. Only one person took part before the study was stopped early.
Phase: PHASE1 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 26, 2026 05:28 UTC
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Scientists track rare genetic disease to unlock its secrets
Knowledge-focused TerminatedThis study looked at people with MECP2 duplication syndrome, a rare genetic condition. Researchers measured changes in biomarkers, symptoms, and quality of life over time. No treatment was given. The goal was to better understand how the disease progresses.
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 22, 2026 13:53 UTC