A decades-old sleeping sickness drug (DFMO) shows surprising promise treating Bachmann-Bupp syndrome, an ultra-rare genetic disorder in children.
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A forgotten drug is giving new hope to kids with a rare disease
DFMO (Decades-Old Sleeping Sickness Treatment) Drug repurposed
Bachmann-Bupp Syndrome (BABS) - Ultra-Rare Genetic Disorder Target disorder
Early Treatments Show Encouraging Results In Handful Of Pati Clinical status