Catabasis Refutes MRI T2 Biomarker In Duchenne Study
Catabasis says biomarker MRI T2 doesn't work after all in boys with Duchenne's, plans to stick with four-step climb test or 10-meter walk/run test from now on.
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Repeated trial failures in this rare disease space have given birth to new alliances – one backed by Critical Path and the other by the CureDuchenne advocacy group – that aim to model disease progression much more accurately through data sharing and rigorous analysis.
Using its safely metabolized and rationally targeted (SMART) linker platform, Catabasis has identified a NF-κB inhibitor – edasalonexent – as a potential Duchenne muscular dystrophy (DMD) treatment. Speaking at the 2017 Biotech Showcase, Dr Jill Milne, co-founder, president and CEO of Catabasis, describes the company’s ambitions to pursue a pivotal Phase III trial this year.
Pink Sheet’s Drug Review Profile looks at US FDA clinical memos opposing approval of Sarepta's Duchenne muscular dystrophy drug eteplirsen, revealing challenges patient advocates and agency face in transforming patient experiences into hard data that can be used for approvals.