The FDA wouldn’t review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.
FDA’s ‘ivory tower thinking’ ignores promising biomarkers, says a rare disease CEO
The FDA wouldn’t review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.
