187: An Interview With Dr. Pradeep P.A. Mammen on Females With Duchenne Muscular Dystrophy

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lisa Shea, director of global patient advocacy and engagement for immunology at Johnson & Johnson, about the experiences of patients affected by HDFN and FNAIT.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Nicole Lamanna, MD, a leukemia physician specializing in CLL treatment at Columbia University Medical Center in New York City.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews May Lee Tjoa, PhD, senior global medical affairs leader for Johnson & Johnson’s nipocalimab and maternal-fetal immunology division.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Mascarenhas, MD, a professor with the Icahn School of Medicine at Mount Sinai in New York, on advancing care in myelofibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Catherine Miller, PharmD, global medical affairs lead for hereditary angioedema (HAE) at Intellia Therapeutics, on an experimental gene editing therapy known as lonvo-Z.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Shoshana Revel-Vilk, MD, director of the Gaucher unit at Shaare Zedek Medical Center in Jerusalem, Israel.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Nikki McIntoch, mother of a boy with spinal muscular atrophy (SMA) and founder of the advocacy group Rare Mamas.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Brown, MD, PhD, director of the CLL Center at Dana-Farber Cancer Institute in Boston, Massachusetts, and winner of the 2025 Michael J. Keating Outstanding Achievement Award.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews neurologist Michio Hirano, MD, of Columbia University in New York about an investigative therapy that shows promise in treating thymidine kinase 2 deficiency (TK2d), an ultrarare disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews cardiologist Pradeep P.A. Mammen, MD, of the University of Kansas School of Medicine, on the growing awareness of girls affected by Duchenne muscular dystrophy.