Feb. 24 at 3:49 AM
$XBI Love watching the FDA take action to streamline rare and ultra rare disease treatments to patients. I've heard whispers that this would help
$SRPT $CAPR,
$TLSA and
$CADL, but who else do you think would benefit from this movement?
Dr. Marty McKary (FDA Commissioner; posted on X):
https://x.com/DrMakaryFDA
"The FDA has a moral duty to move quickly to get meaningful treatments to patients."
"Collectively, 30 million Americans have a rare disease. The problem isn't rare — it's a call to action."
"I’ve signed 100% of compassionate use requests that have come to my desk."
Exciting times folks.
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