@DrMakaryFDA Then why do you keep blocking access for children with MPS II AND IIIA to 3 drugs proven safe and effective through years long trials with a scientifically proven biomarker. The ultra rare community will no longer stand by and wait for our children to die.
@DrMakaryFDA Walk the walk! Rare Disease kids are suffering/dying because the @US_FDA is not using their authority and flexibility given to them by congress! Accelerated approval is not being used.