DFMO Repurposed for Bachmann‑Bupp Syndrome in Kids
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- DFMO – the drug, also known as eflornithine, originally approved for West African sleeping sickness and facial hair reduction, is now being used to inhibit the overactive ODC1 enzyme in BABS patients.
- Corewell Health – pediatric geneticist Dr. Caleb Bupp leads the clinical effort and coordinates the FDA‑approved single‑patient investigational protocol for DFMO in BABS.
- Every Cure – the nonprofit biotech group provides regulatory navigation and compliance support, accelerating the trial’s progress and expanding patient access.
- FDA – granted a single‑patient investigational protocol allowing DFMO to be administered to BABS patients outside of standard clinical trials.
- Michigan State University – its College of Human Medicine contributed preclinical studies and retrospective analyses that identified DFMO’s mechanistic fit for ODC1 gain‑of‑function mutations.
Why it matters: Not enough in the article to say why this matters.
