Santhera's Duchenne effort gets a boost on positive PhIIa for alternative steroid vamorolone — though there's a lot left to prove
Months after Santhera got its hands on a second Duchenne muscular dystrophy drug, the Swiss company is getting an early look at how it improves muscle function. But serious questions remain as it takes on a field stuffed with entrenched rivals and controversy.
ReveraGen was in charge of the Phase IIa extension, which compared different doses of vamorolone with natural history controls. With 12 boys (aged 4 to 7) in each of four treatment groups, investigators tested 0.25, 0.75, 2.0 and 6.0 mg/kg/day in the study and published the results in Neurology.
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