Ionis Pharmaceuticals, Inc.
Clinical trials sponsored by Ionis Pharmaceuticals, Inc., explained in plain language.
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Early safety trial of ION269 for Alzheimer's in down syndrome ends after just one participant
Disease control TerminatedThis early-stage study tested a drug called ION269 in adults with Down syndrome who also showed signs of Alzheimer's disease. The goal was to check if the drug was safe and tolerable when injected into the spinal fluid. Only one person took part, and the study was stopped early, …
Phase: PHASE1 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Scientists hunt for clues in rare genetic brain disorder
Knowledge-focused TerminatedThis study aimed to observe how MECP2 duplication syndrome progresses over time by measuring biological markers in spinal fluid and blood, and by tracking changes in development, behavior, and seizures. It enrolled 29 males aged 1 month to 65 years with a confirmed genetic diagno…
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC