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Rare Care Podcast

Dec 29, 2022

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Philip J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at NCATS. Dr. Brooks describes the mission of the Bespoke Gene Therapy Consortium, which recently marked its first anniversary.


Dec 9, 2022

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Scott Santarella, the new president and CEO of the Miami-based Alpha-1 Foundation—the nation's largest organization working on behalf of patients with alpha-1 antitrypsin deficiency (AATD).


Dec 5, 2022

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lourdes Rocha-Nussbaum, director of the Cholangiocarcinoma Foundation's Veterans Project. This program explores the relatively high incidence of bile duct cancer among Vietnam War veterans and care options for such patients.


Nov 18, 2022

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Darlene Shelton, founder and president of Danny's Dose Alliance, a Missouri-based nonprofit that seeks to ensure proper emergency medical care for all patients with hemophilia and other bleeding disorders.


Nov 16, 2022

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Aiwu Ruth He, MD, PhD, an associate professor of medicine at Medstar Georgetown University Hospital in Washington, D.C., and one of the principal investigators of the TOPAZ-1 clinical...