The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Sarepta’s gene therapy for Duchenne muscular ... In particular, it highlighted “compelling” improvements over placebo on secondary endpoints like improvements in time to rise from the ...

Lumin Digital, a leading cloud-native digital banking provider, today announced that clients have invested over $75 million into the business, highlighting a dedication to partnership in which clients ...

There were, however, significant improvements on secondary measures, including time to rise and a 10-metre walk test that, according to Sarepta, were “evidence of a clinically meaningful benefit ...

A key secondary endpoint, change from baseline in ... Some of its programs - like DMD and HCM - have strong competition, from Sarepta and BMS, and also from others. This may not be a major risk ...