Not sure if my math is correct, but in the US alone there is roughly 1.9 million girls born per year. Rett prevalence is 1 in 10,000, so about 190 girls per year are afflicted with this awful disease. If AVXL can treat 1 - 20 year old girls, that means roughly 4,000 girls could benefit currently. In comparison, for Sarepta, DMD affects 1 in 3500 boys. However, the first indication was for Exon 51 DMD which was 13% of DMD boys, or about 1 in 27,000 (about 70 boys per year). These rare diseases are devastating and drugs like 273 are so critical to their target communities.....thank goodness they are are being addressed by companies like Anavex.
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