Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Peter Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), on the 40th anniversary of the landmark Orphan Drug Act.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sara Rothschild, the new executive director of the Life Raft Group, the nation's chief nonprofit advocating on behalf of patients with gastrointestinal stromal tumor (GIST).

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.

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).

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.

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.

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 trial.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Brenda Wong, MD, an expert on Duchenne muscular dystrophy and director of the MDA Care Center at the University of Massachusetts Chan Medical School in Worcester.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Randi Clites, rare disease policy director at the Ohio-based Little Hercules Foundation. This nonprofit's mission is to advocate for patients with Duchenne muscular dystrophy (DMD) and fight to get DMD included on every state's newborn screening panel.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sumaira Ahmed, founder and executive director of The Sumaira Foundation, a Boston-based nonprofit organization that advocates on behalf of patients with neuromyelitis optica spectrum disorder (NMOSD) and related diseases.