Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donna Appell, founder and executive director of the Hermansky-Pudlak Syndrome Network. Patients with the most common type of this rare disease always develop pulmonary fibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Rich Savino, a New Jersey grandfather of a boy with Duchenne muscular dystrophy. Savino, 71, is among a growing number of grandparents becoming advocates for patients affected by DMD.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kimberly Ku, MD, an oncologist with Illinois Cancer Center and host of the podcast "Val-You Based Care,"

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Christine Clemson, PhD, senior vice-president and global head of medical affairs at Entrada Therapeutics, about exon skipping therapies in Duchenne.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Avril Daly, president of Paris-based Eurordis-Rare Diseases Europe, on how the EU is falling behind in clinical trials for rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jim Palma, CEO of the TargetCancer Foundation, a nonprofit organization that focuses on finding treatments for rare cancers.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Duchenne expert Paul Esteso, MD, PhD, medical director of the cardiac antithrombosis management program at Boston Children's Hospital.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Teresa Barnes, founder of PF Warriors—a global support network that provides education, inspiration and hope to individuals and families affected by pulmonary fibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Martina Cornel, MD, PhD, about the importance of adopting standardized newborn screening for rare diseases throughout Europe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Diane Zipursky Quale, who founded the Bladder Cancer Advocacy Network 20 years ago with her late husband after he developed bladder cancer.