Dossier
DFMO
Coverage of DFMO in the Nexus archive.
- A forgotten drug is giving new hope to kids with a rare disease
A decades-old drug, DFMO, originally used to treat sleeping sickness, is showing promise in treating Bachmann-Bupp syndrome (BABS), an ultra-rare genetic disorder. Early results indicate it may alleviate severe symptoms by addressing the underlying genetic malfunction, though regulatory and logistical challenges slow progress.