Dawn Phillips

Dawn Phillips


Job title: Senior Director Clinical Outcomes Research


Challenges in Clinical Outcome Selection & Validation in Neuromuscular Gene Therapy 11:15 am

The impact of accelerated approvals on clinical outcome considerations Defining outcomes that are likely to predict clinical benefit Exploring the relationship between functional measures and clinical biomarkers Navigating the heterogeneity of neuromuscular diseases and how this impacts clinical endpoints and control variables Leveraging natural history data to inform study design considerations and responder definitions Administrative…Read more

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