Drisapersen Advisory Committee Is Rough Welcome To FDA For Patient Groups
Advocates counter FDA’s problems with Duchenne muscular dystrophy treatment's trial data using real-life experience.
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Circumstances of FDA's approval of Sarepta's Exondys 51 approval may give BioMarin reason to appeal the complete response for its Duchenne muscular dystrophy treatment drisapersen – but its high-quality data could preclude success.
Duchenne Group's Presentation Is Milestone For Patient Involvement
Advocacy group gets portion of Sarepta's formal presentation period to present eteplirsen patient experience data, believed to be a first for an advisory committee meeting. Will it become commonplace?
Sarepta's Additional Data Doesn't Sway FDA On Eteplirsen
Agency reviewers raise more concerns about efficacy of proposed Duchenne muscular dystrophy treatment despite new information.