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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 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