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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 malfunction. Researchers have already treated a handful of patients with encouraging results, but progress has been slowed by regulatory and logistical hurdles.
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