Duchenne Development: Data Sharing May Enable Trials To Move Beyond Six-Minute Walk
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.
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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.
‘File Over Protest’ At US FDA: PTC To Pursue Rarely-Used Pathway For DMD Drug
Translarna’s best path forward is the file over protest pathway for the application, which received a refuse to file letter last year, PTC says; former OND Director John Jenkins calls it ‘a fairly rare event.’