240: An Interview With Christine Clemson, PhD, of Entrada Therapeutics

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Rebecca Aune, director of education programs at the National Organization for Rare Disorders (NORD), about Rare Research Launchpad. The new program. The free, self-paced online series–designed to introduce students and early-career scientists to research and career opportunities–uses cystic fibrosis and Krabbe disease as real-world case studies. Hosted by Simplecast, an AdsWizz company. See https://pcm.adswizz.comfor information about our collection and use of personal data foradvertising.

Larry Luxner, senior correspoondent for Rare Disease Advisor, interviews Aarush Shah, a high-school senior who, along with classmate Veer Chandwani, recently established OAI Nexus, the first public AI platform built exclusively for rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Doug Flora, MD, about his new book, "Rebooting Cancer Care: Can AI Make Care More Human Again?"

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.