BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

Limb-girdle muscular dystrophy type 2i is a rare, genetic condition that causes progressive muscle degeneration that can impact skeletal, respiratory, and cardiac muscles. As the condition progresses, people lose the ability to perform routine daily activities, such as walking or standing up without assistance. There are no therapies available today to slow, halt, or reverse the condition. ML Bio, a company founded by two patient families in search of treatments for the condition and later acquired by BridgeBio, is advancing an experimental therapy with the potential to become the first oral treatment for the limb-gridle. We spoke to Doug Sproule, chief medical officer of ML Bio Solutions, about limb girdle muscular dystrophy type 2i, the company’s experimental therapy to treat the condition, and the power of rare disease patient families to shape drug development. One note before we begin. Early in the discussion Sproule misspeaks. The founders of ML Bio are the McColl and Lockwood families and the company’s lead experimental therapy was discovered at the McColl-Lockwood Lab.