Larry Luxner, senior corespondent for Rare Disease Advisor, interviews Ron Bartek, founding president of the Freidriech's Ataxia Research Alliance (FARA), about the hope generated by the recent FDA approval of Reata's omaveloxolone (Skyclarys)—the first-ever therapy to treat this rare neuromuscular disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Miranda Bradnick, the mother of 3 children with Alagille syndrome. Among her biggest concerns: how COVID-19 has changed the lives of her family and others affected by rare disease, 3 years after the World Health Organization declared a pandemic.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jeff Szer, BMedSc, a hematology professor at Australia's Royal Melbourne Hospital and secretary of the UK-based International PNH Interest Group, which is organizing its first-ever scientific symposium on paroxysmal nocturnal hemogloblnuria (PNH).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cheryl Schwartz, Takeda's senior vice-president of US rare disease business, on the recent approval of lanadelumab-flyo (Takhzyro®) for hereditary angioedema.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Berk, MD, director of the Boston University School of Medicine's Localized Amyloid Clinic, on the latest therapeutic options for hereditary ATTR amyloidosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Çem Akin, MD, a specialist in allergy and immunology at the University of Michigan, about various treatment options for systemic mastocytosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dr. Abby Sandler of the National Cancer Institute.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Yale University hematologist George Goshua, MD, who has completed a cost analysis of gene therapy versus standard of care in patients with sickle cell disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Arushi Khurana, MBBS, a hematologist and lead author of a study showing that minorities are less likely to receive front-line therapy for diffuse large B-cell lymphoma than white patients.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Catherine Broome, MD, on the relative benefits to quality of life by treating immune thrombocytopenia patients with efgartigimod.