Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Federico Stella, MD, on the controversial practice of severely restricting the diets of patients who have just undergone stem cell transplants.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abigail Jenkins, president and CEO of Gamida Cell Ltd., on the company's development of omidubicel as an alternative to umbilical cord blood in stem cell transplants for myelofibrosis and other patients.

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.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Martin Mense, PhD, senior vice president of drug discovery at the Cystic Fibrosis Foundation, and head of the CFF's Therapeutics Lab in Lexington, Massachusetts.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Levy, MD, associate professor of neurology at Harvard Medical School in Boston, Massachusetts, and a leading expert on neuromyelitis optica spectrum disorder (NMOSD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Wallace Valdes, PT, founder of the Duchenne Therapy Network and physical therapist with CureDuchenne, on the importance of physical therapy and exercise in boys and young men with Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Janet Lynch Lambert, CEO of the Alliance for Regenerative Medicine (ARM). This Washington, DC-based nonprofit aims to bring safe and effective cell and gene therapies to patients around the world.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Annie Kennedy, head of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases. This Washington, DC-based nonprofit focuses on eliminating the diagnostic odyssey for rare disease, promoting regulations that speed up therapeutic development, and improving access to approved rare-disease therapies.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Kelly, PhD, chief scientific officer at CureDuchenne, on the latest developments in exon skipping, gene therapy and other treatments for Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eileen Sullivan Baker, an Ohio attorney whose son, John, has long chain fatty acid oxidation disorder. She has become an outspoken advocate for LCFAOD, in the absence of any formal patient advocacy organization for those with the disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul Heaton, a Montana cattle rancher whose son has Duchenne, on his Calves2Cure DMD fundraising initiative.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Emil Kakkis, MD, PhD, founder and CEO of San Francisco-based Ultragenyx, on the increasingly complex challenges facing developers of drugs to treat rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Pradeep Mammen, MD, professor of internal medicine and director of the Neuromuscular Cardiomyopathy Clinic at UT Southwestern in Dallas, Texas. Dr. Mammen, a speaker at the CureDuchenne 2022 Futures conference, has done extensive research on the issue of female carriers of muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews wheelchair-bound Justin Skeesuck, who didn't let his progressive genetic disease stop him from crossing Spain's 500-mile Camino de Santiago. The adventure, with his best friend Patrick Gray, led to the book and movie "I'll Push You." Justin and Patrick were keynote speakers at the recent CureDuchenne 2022 Futures conference in...