But this all is pretty encouraging -- especially given the high overall burden -- of these brain-devouring diseases. Here's the latest:
. . .The dazzling 36-month clinical data announced by uniQure (NASDAQ: QURE) showing a significant slowing of Huntington’s disease (HD) progression in patients treated with its gene therapy candidate AMT-130 was just one of several reasons why the company’s stock more than tripled this past week on the better-than-expected news, reaching a five-year high.
Part of the. . . buying from investors reflected the strikingly positive tone taken by investigators—and analysts—in discussing the results. Key among those results was the statistically significant 75% slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients treated with the high dose of 6×1013 gc/subject, meeting the primary endpoint of the study.
“[A] 75% slowing of disease progression is greater than what we even anticipated, expected, and hoped for. That means for one year of disease progression, they will have four years longer in terms of disease-free life,” Sarah Tabrizi, MD, PhD, director of the University College London (UCL) Huntington’s Disease Centre, told analysts Wednesday on a conference call convened by uniQure to discuss the data. “The effect of 75% slowing is a huge effect size and will have massive effects for patients’ lives. . . .”
Clearly I am scouring high- and low-, to find good news [and look away from the chaos Tangerine has created -- but does not even remotely understand -- in Iran, and beyond -- in the Strait of Hormuz]. Onward, just the same.
नमस्ते
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