(Reuters) -BridgeBio Pharma said on Monday its experimental drug for a rare muscle disorder, which currently has no approved treatments, showed improvements in motor and lung function in a late-stage trial.
The oral drug, BBP-418, is being tested in patients with a form of limb-girdle muscular dystrophy, or LGMD, a group of inherited genetic disorders characterized by progressive muscle weakness and wasting.
LGMD has an estimated global prevalence ranging from 1 to 6 per 100,000 people, with about 5,000 people in the U.S. currently living with the condition.
BBP-418 met the main goal of significantly increasing glycosylated αDG, a key marker of LGMD, by 1.8 times from baseline when compared to placebo in an interim analysis of the trial at three months.
Glycosylated αDG is a form of th

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