TAY SACHS DISEASE
Clinical trials for TAY SACHS DISEASE explained in plain language.
Never miss a new study
Get alerted when new TAY SACHS DISEASE trials appear
Sign up with your email to follow new studies for TAY SACHS DISEASE, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Scientists test gene therapy for devastating childhood brain disease
Disease control TerminatedThis early-stage study tested the safety of an experimental gene therapy for children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that destroy the brain and nervous system. The therapy aimed to deliver working copies of the faulty genes directly into the …
Matched conditions: TAY SACHS DISEASE
Phase: PHASE1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
-
Race against time: new pill tested to halt rare brain disease
Disease control TerminatedThis study tested whether a daily oral pill called venglustat could slow the progression of late-onset Tay-Sachs and Sandhoff disease, rare and severe inherited disorders that damage the brain and nerves. It involved about 75 adults and some younger participants over a two-year t…
Matched conditions: TAY SACHS DISEASE
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC