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Orphan Drug Congress Europe | Booth #54

Meet Veristat at the Orphan Drug Congress 2021 taking place on 15-18 November 2021, Booth #54

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Veristat Talk at the Congress

Wednesday 17th November/ 11.40 a.m

" The Role of Natural History Studies in Ultra-Rare Disease Trials– Takeaways from a Canavan Disease Gene Therapy Trial on Their Challenges, Benefits and Successes"

Speakers:

John Balser, PhD, President and Chief Biostatistician, Veristat

Rachel Smith, Portfolio Director, Veristat

Abstract:

Completing a natural history study in conjunction with an interventional study for ultra-rare diseases can be critical to understanding the disease and its progression. The data from the natural history can be essential to informing the design and outcome selection of the interventional study. We explore the challenges of setting up a natural history study with both retrospective and prospective data to develop a new rating scale for disease progression in Canavan disease, a rare and relatively unknown childhood disease.

Our experts will share their experience, successes, and lessons learned on :