Rare Care Podcast

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.