Heart inflammation emerges in a Sarepta Duchenne patient, clouding an otherwise positive readout
Having already secured several approvals for Duchenne muscular dystrophy drugs, Sarepta aimed to capitalize on its lead in the field with a new data readout Wednesday. And while many of the hard numbers are positive, a new side effect is putting a damper on the mood.
Sarepta reported Wednesday that its experimental gene therapy, dubbed SRP-9001, significantly improved patients’ motor function after one year compared to a control. The data came from a 20-patient cohort of boys aged four to seven years old and also passed several key secondary tests. The biotech also revealed four-year data from a small, four-patient cohort in an earlier study suggesting the treatment’s effect can remain durable over that time.
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