The disease natural history is a comprehensive description of the disease without any interventions. Establishing this history is a vital step toward effective clinical trials because the natural history of the disease provides objective, quantifiable ways to definitively say whether a potential therapy is having the desired affect.
The natural history of infantile Tay-Sachs and Sandhoff is expected to be published later in 2011. This study was the result of 100 families' having completed detailed questionnaires about their child’s development, loss of skills and disease progression.
Currently, researchers are recruiting children affected by Tay-Sachs, Sandhoff and GM-1 for additional natural history studies following their disease course over one year. Contact the office at 1-800-906-8723 or
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to learn more.
A Canavan natural history study is currently ongoing in Europe.
It takes the right combination of passionate researchers and funding to actively pursue these vitally important but non-therapeutic studies.
