FDA’s new leader must keep agency’s promises to rare disease patients - Las Vegas Sun News
• Following the resignation of Commissioner Marty Makary, the FDA is seeking a new leader to oversee the agency's commitment to accelerating treatment access for rare disease patients. • The urgency is highlighted by UX111 gene therapy, which clinical trials show can reduce toxin buildup for over eight years and slow cognitive decline if administered early. • This transition is critical because children unable to access UX111 through clinical trials continue to suffer irreversible losses while awaiting formal FDA approval.
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