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March 17, 2021 33 min
Seven thousand rare diseases, affecting 1 in 10 -Americans. With 93% of these diseases without an FDA-approved treatment. And With 50% of all rare disease patients are children. In this episode of Physician's Weekly, Peter Hofland, Ph.D. talks with Marissa Penrod, the founder and Chief Executive Officer of Team Joseph, a nonprofit organization dedicated to funding research for Duchenne Muscular Dystrophy, and Steve Smith, who serves on the advisory committee of RareDisease Legislative Advocates, a program of the EveryLife Foundation and is the father of a son with a rare disease.

Also in this episode:
- Rachel Giles, MD. talks with Tom Powels, MBBS, MRCP, MD, Professor of Genitourinary Oncology,
Director, Barts Cancer Centre, and Lead for Solid Tumour Research about how to design good clinical trials;
- Paul Schmidt talks with Adam J. Ratner, MD, MPH, Associate Professor at the Department of Pediatrics and the Department of Microbiology at NYU Grossman School of Medicine and Director of the Division of Pediatric Infectious Diseases about pediatric vaccines and COVID-19.
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