EP 21 Fikri Birey

Pioneering Genetic Therapies for Rare Disorders | Dr. Fikri Birey | Rare Awareness Radio Description: In this powerful episode of Rare Awareness Radio, we sit down with Dr. Fikri Birey — neuroscientist, stem cell researcher, and Assistant Professor at Emory University — whose groundbreaking work is advancing the understanding and treatment of rare neurological disorders, including Timothy Syndrome and CACNA1A-related channelopathies. Dr. Birey shares his journey from Cyprus to Stanford to Emory, where he now leads a lab focused on the use of patient-derived 3D brain organoids to model disease at the cellular level. We explore how these human cellular models are helping scientists investigate the underlying mechanisms of rare disorders and test novel therapeutic approaches, including CRISPR and antisense oligonucleotides (ASOs). He also reflects on the critical role of patient advocacy groups like the CACNA1A Foundation, the urgency of translational research, and why collaboration across disciplines is essential to accelerating cures. Whether you're a patient, caregiver, scientist, or simply curious about the future of genetic medicine, this episode offers hope, insight, and inspiration. 🔬 Topics Covered: What are calcium channelopathies? 3D brain organoids and assembloids Timothy Syndrome and CACNA1A research Genetic therapeutics and personalized medicine Advice for rare disease families Role of advocacy groups in shaping research 📢 Don’t forget to like, share, and subscribe for more conversations that raise awareness, foster connection, and accelerate impact in the rare disease community. 🎧 Listen on all major podcast platforms.