From Lab to Clinical Trial: Rare Disease and Newborn Screening


Derek Ansel | Amy Gaviglio - Public Health Genetics and Genomics Consultant; Anthony Steyermark - Medical Director | The Legacy of Angels Foundation

In this webinar, our speakers talk about how newborn screening (NBS) has helped the rare disease community and the potential for closer partnership in clinical trials. Topics will include:
• History and purpose of NBS in the US and globally
• Diagnostic journey for rare disease patients and the role of NBS
• NBS public policy
• Challenges and limitations of screening programs and addition of more panels
• Advocacy efforts for NBS
• Why NBS is important for enrollment of rare disease clinical trials

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Derek Ansel
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Derek Ansel

Senior Director, Therapeutic Strategy Lead, Rare Diseases
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