BridgeBio’s long-term limb-girdle muscular dystrophy data; QHP buys a CDMO
2023-10-09
临床2期临床结果临床3期
BridgeBio shares longer-term PhII data in limb-girdle: BridgeBio said that patients with limb-girdle muscular dystrophy type 2I/R9 treated with BBP-418 saw “stabilization in NSAD scores and ambulatory measures” over 21 months in the 14-patient Phase II study. The company is already running a Phase III study that it hopes to use for an accelerated approval of the limb-girdle treatment, with glycosylated α-dystroglycan levels at 12 months as a surrogate endpoint. The confirmatory results from the study would be a clinical test for limb-girdle dystrophies called the North Star Assessment for Dysferlinopathy, or NSAD. — Lei Lei Wu
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来源: EndPoints
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