Leading the Fight to treatand cure Tay-Sachs, Canavan and related diseases
Research is making exciting advances towards a treatment and a cure. It is important to note that Tay-Sachs research is meaningful to Sandhoff and GM-1 as these diseases share many biochemical traits.
Researchers have cured Tay-Sachs mice and greatly improved the condition of Tay-Sachs cats. Plans are currently underway to approach the FDA for approval of a Phase I clinical trial. Moreâ€¦
Pyrimethamine (PYR) molecular chaperone is currently in clinical trials of LOTS. At the right dose PYR could increases
enzyme activity. Moreâ€¦
substrate inhibition clinical trial was not considered a success and it is not currently being pursued as a treatment, but the drug is available for off-label use. Many families felt the drug slowed the disease progression. Moreâ€¦
Exciting breakthrough work on the potential therapeutic uses of stem cells was done in Sandhoff mice. Stem cell therapy is still an emerging field; potential therapies or clinical trials using stem cell therapy are not immediate. Moreâ€¦
Bone Marrow Transplant is an extremely invasive and experimental intervention. Moreâ€¦
National Tay-Sachs & Allied Diseases Association2001 Beacon StreetSuite 204Boston, MA 02135
email@example.com(617) 277-4463 phone(617) 277-0134 fax
Biotechnology Info www.ncbi.nlm.nih.gov
At NTSAD, we are making a difference. Every day. Helping families and individuals find their way and providing hope by supporting research on many levels. All of this takes compassion and determination.
There is no time to waste, and families need our help. Those who are fighting today and those who will seek our help tomorrow.
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