Medicine
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UCI Health treated the first patient in REGEN4HD, moving hNSC-01 from preclinical promise into human safety testing for Huntington's disease.
Why now
First-patient dosing moves a neural stem cell strategy for Huntington's disease from preclinical story to monitored human safety trial.
Riaan Singh Digeorge received an experimental AAV9 gene therapy after a parent-led rare-disease development effort raised millions and reached an FDA-cleared IND.
Why now
A parent-led effort moved an ultra-rare disease from no approved treatment to first-in-human dosing, making the development story as important as the science.
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