Tuesday, March 28, 2023 10:23:26 AM
It is estimated that between 2 and 6 people per 100,000 have the disease. At this rate, with 330,000,000 Americans, there would be between 6,600 and 19,800 cases.
A certain number, probably a majority, are likely to be like mine; are confounding and disagreeable, but not severe enough to require treatment.
But where blarcamesine will be transformative will be for what are called "complicated" cases of HSP. Those are the ones that appear early in a child's life, and severely affect muscles across the entire body, not just the muscles controlling gait. Complicated HSP is eventually lethal, but the child suffers unbelievably, often for many years, often suffering to die in his or her second decade. Just horrible.
So, for complicated cases of HSP, infrequency of the disease should not inhibit therapy. It's a classic "rare disease." Blarcamesine needs to be tested for it.
Comprehensive info on the disease here:
https://en.wikipedia.org/wiki/Hereditary_spastic_paraplegia
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