Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

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Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i

The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for limb-girdle muscular dystrophy type 2i (LGMD2i). BBP-418 is a small molecule that delivers a prodrug — a precursor molecule — which, once inside the body, will change into the sugar component of alpha-dystroglycan. The lack of a working alpha-dystroglycan causes muscle cells of LGMD2i patients to lose their structure, leading to muscle weakness and disability. Also called…

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