How RNA Therapies Are Changing Duchenne | Dr. Yoshitsugu Aoki

Dr. Yoshitsugu Aoki is a physician-scientist at the forefront of RNA therapeutics and genetic medicine.As Director of the Department of Molecular Therapy at the National Center of Neurology and Psychiatry in Tokyo, Dr. Aoki has helped lead the development of antisense oligonucleotide (ASO) therapies for Duchenne muscular dystrophy—including viltolarsen, an exon-skipping therapy approved in both Japan and the United States.In this conversation, we explore how RNA therapies became a reality, what they’re doing for patients today, and where the field is heading next.⸻🔬 What we cover:What exon skipping is (in simple terms)How ASO therapies are helping Duchenne patientsBreakthrough clinical results and what they meanDelivering therapies to the brainThe future of RNA, gene therapy, and combination treatmentsHow AI and robotics could accelerate drug development⸻🌐 GENEration HopeI’ve launched the new GENEration Hope website along with a newsletter and weekly news updates covering the latest breakthroughs in genetic medicine.👉 Visit: generationhope.co👉 Subscribe to stay up to date on what’s coming nextIf you find this valuable, liking and subscribing really helps support the project.⸻⚠️ DisclaimerThis content is for informational purposes only and is not medical advice.