SANDHOFF DISEASE
Clinical trials for SANDHOFF DISEASE explained in plain language.
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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: SANDHOFF DISEASE
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Scientists test gene therapy for devastating childhood brain disease
Disease control TerminatedThis early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that destroy the brain. The study aimed to see if delivering working copies of the faulty genes directly into the brain and spinal flui…
Matched conditions: SANDHOFF DISEASE
Phase: PHASE1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated Mar 02, 2026 15:27 UTC