George Lee

Thank you, @FDA_KyleD and @FDA, for recognizing the urgency facing rare disease families and advancing a framework that reflects the complexities of diseases like Huntington’s disease. The HD community has contributed decades of natural history data, clinical trial participation, and documented risk tolerance. We welcome Priority Review of AMT-130’s BLA, but continue to advocate for CNPV consideration. If a promising gene therapy for a fatal, relentlessly progressive disease does not warrant consideration for every appropriate expedited pathway, what does? The success of regulatory reform will be measured not by the hope it promises, but by the patients it reaches while there is still time. Our community has done its part. Now we look to the FDA’s leadership to ensure that urgency is translated into meaningful action. 💙💜 #HuntingtonsDisease #RareDisease