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Hope for Rare BABS: DFMO Trials Approved by FDA

1/16/2026

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Summary

Story summary

Bachmann-Bupp syndrome (BABS) is a rare genetic disorder with no established therapy; researchers are evaluating difluoromethylornithine (DFMO) as a potential treatment.
A collaboration among Corewell Health, Michigan State University, and Every Cure aims to navigate regulatory challenges and advance clinical trials of DFMO for BABS.
The U.S. FDA has encouraged the team to proceed with a clinical trial, and they plan a preclinical study next year.

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Story summary

Bachmann-Bupp syndrome (BABS) is a rare genetic disorder with no established therapy; researchers are evaluating difluoromethylornithine (DFMO) as a potential treatment.
A collaboration among Corewell Health, Michigan State University, and Every Cure aims to navigate regulatory challenges and advance clinical trials of DFMO for BABS.
The U.S. FDA has encouraged the team to proceed with a clinical trial, and they plan a preclinical study next year.