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.
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?
Agency reviewers raise more concerns about efficacy of proposed Duchenne muscular dystrophy treatment despite new information.