Leading the Fight to treat and cure Tay-Sachs, Canavan and related diseases
This approach is based on the theory that the
myelin destruction seen in Canavan is caused by low levels of acetate. The deficient or missing
enzyme in Canavan is responsible for breaking down N-acetylaspartate acid into acetate. By supplementing acetate normal myelin production and maintenance can occur but does not address the accumulation of N-acetylaspartate acid.
Canavan animal model studies have been very promising. Two infants in Israel received acetate supplement for several months. It was well tolerated but did not show clinical improvement. Learn more about the study at Science Direct. Acetate is included in a regimen of medications prescribed to Canavan children on a case by case basis. Contact our director of family services, Kim Kubilus at
or 800.906.8723 to learn more.
Scientists are still learning about Canavan disease mechanism. Some scientists do not agree with the theory that the myelin destruction seen in Canavan is caused by low levels of acetate. It is also not well understood how the body absorbs and metabolizes acetate and if it passes the blood brain barrier into the
central nervous system.
National Tay-Sachs & Allied Diseases Association2001 Beacon StreetSuite 204Boston, MA 02135
firstname.lastname@example.org phone617-277-0134 fax
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