Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dr. Haggi Mazeh, an endocrine surgeon at Hebrew University's Hadassah Medical Center in Jerusalem. Dr. Mazeh, one of Israel's top experts on thyroid cancer, talks extensively about the challenges of treating medullary thyroid carcinoma.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Harsha K. Rajasimha, PhD, in Herndon, Virginia. Dr. Rajasimha cofounded the Organization for Rare Diseases India and is now CEO of Jeeva Informatics, which aims to improve the diversity, equity, and inclusion (DE&I) of patients in clinical trials worldwide.

Larry Luxner, senior correspondent for Rare Disease Advisor, talks to pulmonologist Naftali Kaminski, MD, professor of medicine and pharmacology and chief of Pulmonary, Critical Care and Sleep Medicine at Yale University School of Medicine in New Haven, Connecticut. Dr. Kaminski discusses the recently announced Three Lakes Consortium for Pulmonary Fibrosis (TLC4PF), a consortium involving the Thre...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Durhane Wong-Rieger, PhD, president and CEO of the Canadian Organization for Rare Disorders (CORD) and chair of Rare Diseases International (RDI). Dr. Wong-Rieger talks about the challenges of diagnosing and treating diseases in Canada, as well as advancing access to therapies around the globe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews David Goldenberg, MD, FACS, a surgical oncologist, and professor and chair of the department of otolaryngology-head and neck surgery at Penn State College of Medicine in Hershey, Pennsylvania. Dr. Goldenberg has just published the latest edition of his book, Endocrine Surgery of the Head and Neck, a significant portion of whic...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Doug Kerr, MD, PhD, chief medical officer at Generation Bio in Cambridge, Massachusetts. The company is aggressively pursuing redosable gene therapies for hemophilia and other rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jerry Vockley, MD, PhD, chief of medical genetics at Pennsylvania's UPMC Children's Hospital of Pittsburgh. Dr. Vockley is also the founder of INFORM, the International Network for Fatty Acid Oxidation Research and Management, which will hold its next annual conference virtually Oct. 27-28.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Hagit Baris-Feldman, MD, director of the Genetics Institute at Israel's Tel Aviv Sourasky Medical Center. Dr. Baris-Feldman, who also chairs the Israel Medical Genetics Association and is an associate professor at Tel Aviv University's Sackler Faculty of Medicine, talks about why Israel has emerged as a global leader in the di...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Barry Byrne, MD, director of the University of Florida's Powell Gene Therapy Center in Gainesville. Dr. Byrne has spent years researching potential gene therapies for Pompe disease, comments on the FDA's recent approval of avalglucosidase alfa-ngpt (Nexviazyme) to treat Pompe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Olson, PhD, chief scientific advisor at Vertex Pharmaceuticals and founding chair of the molecular biology department at the University of Texas-Southwestern Medical Center. Dr. Olson is an expert on Duchenne muscular dystrophy and CRISPR gene editing technology.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Edward Neilan, MD, chief medical officer at the National Organization for Rare Disorders (NORD). This is a new position at NORD, which serves as the umbrella organization for some 330 disease-specific patient advocacy groups. NORD also sponsors the annual Rare Diseases and Orphan Products Breakthrough Summit, which this year t...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Peter Knox, senior research director at the Life Raft Group. Based in Wayne, New Jersey, this organization was incorporated in 2002 and advocates for people with all forms of gastrointestinal stromal tumor, or GIST.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Camille Bedrosian, MD, chief medical officer and executive vice president of Ultragenyx Pharmaceutical in Novato, California. Earlier this year, Ulragenyx won approval from the US Food and Drug Administration (FDA) for triheptanoin (Dojolvi®), a liquid medication that supplies medium-chain, odd-carbon fatty acids as an energy...