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BridgeBio Pharma Inc (NASDAQ: BBIO) and affiliate ML Bio Solutions have dosed the first patient in the Phase 2 trial evaluating BBP-418 in patients with Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i).
LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP), leading to chronic muscle injury and loss, muscle weakness, and disability.
BBP-418 is designed to bypass the metabolic defect in LGMD2i by providing the FKRP enzyme with precursor sugar molecules that supplement the body's natural sugars used by FKRP to glycosylate the αDG protein on muscle cells.
The 16-subject trial will assess the safety and key efficacy measurements, including changes in muscle αDG glycosylation levels and changes in functional measures, including a 10-meter walk and North Star for Dysferlinopathy.
Price Action: BBIO increased 4.53% at $66.7 in the market trading hours on last check Friday.
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