The CheckRare Brief delivers a concise summary of the week’s most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.
Madaline Spencer, Podcast Producer; James Radke, PhD, Education Director; Peter Ciszewski, Founder and CEO, CheckRare.
References for this episode’s topics:FDA Advisory Report on Capricor Therapeutics’ Orphan Drug to Treat Cardiomyopathy in Duchenne Muscular DystrophyFDA Advisory Board Report: Link Capricor’s response: Link Cognitive Decline in Immune ThrombocytopeniaInterview with Dr. David Kuter: Link August is SMA Awareness MonthSMA Awareness Page: Link
The CheckRare Podcast Network is dedicated to delivering news, education, and expert insights across the rare disease community. From physician interviews and conference coverage to weekly news updates and patient stories, our family of podcasts connects healthcare professionals, researchers, advocates, industry leaders, and patients with the information that matters most. Produced by CheckRare, each series is designed to advance awareness, education, and clinical care in rare diseases.