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The Future of Genetics in Pediatric Care with Dr. Caleb Bupp

FYI - For Your Innovation

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The Polyamine Pathway: A Novel Drug Repurposing Treatment

The drug eflornatine or Dfmo has been around since the 70s. It was developed as an anti cancer drug but it works really well for West African sleeping sickness. The WHO had kind of kept it around for years and years. So what we got from that was safety data in kids. We were able to go to the FDA and ask them for compassionate use, I and D investigational new drug approval to try this treatment. And so our patient came in. She didn't roll, she didn't talk, she essentially couldn't do anything. But about a month later her family sent me a picture. A couple of days before Christmas and she hit sprouted eyebrows

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