Somebody they met early during the course of their disease already died at the age of 15. Her daughter's now 16. Once again, the gene therapy that had been available to them. The FDA has just sent them what they call their complete response letter, which means no, you can't do it anymore. And it seems on some pretty flimsy excuses. The the $200 million brand new manufacturing facility apparently had stains on the floor and some cracks on the floor. (04:41–04:48)
CLAIM
Asserts that the FDA sent a complete response letter denying access to a gene therapy.
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