Science2026-05-01
A forgotten drug is giving new hope to kids with a rare disease
A decades-old drug once used to treat sleeping sickness is now showing surprising promise against an ultra-rare and life-threatening genetic disorder called Bachmann-Bupp syndrome (BABS). Early patient treatments suggest the drug, DFMO, may ease severe symptoms by targeting the underlying genetic ma
By Science Daily

A decades-old drug once used to treat sleeping sickness is now showing surprising promise against an ultra-rare and life-threatening genetic disorder called Bachmann-Bupp syndrome (BABS)
Early patient treatments suggest the drug, DFMO, may ease severe symptoms by targeting the underlying genetic ma