Maxwell, age 8, made history this fall when he received a new #GeneTherapy targeting his #UltrarareDisease. Learn more about how researchers created and administered the AAV9 therapy targeting SLC6A1, advancing #PrecisionMedicine for kids who previously could not be treated: https://bit.ly/4rBASR1

At #WODC, Ralf-Dieter Hilgers, RealiseD Coordinator from Sigmund Freud Private University, presented the paradigm shift RealiseD is leading in #UltraRareDisease research. 💡This will take shape in Playbooks—practical guides for better, fairer clinical trials. #RareDisease #RealiseD #OrphanDrugs

🎙️Rare Care: The Rare Disease Advisor Podcast

Larry Luxner, interviews neurologist Michio Hirano, MD, of @columbiauniversity.bsky.social about an investigative therapy that shows promise in treating #TK2d, an #UltrarareDisease

🎧 Listen to the full podcast here: https://bit.ly/4pfywX7

#RareDisease

Joint, multifaceted genomic analysis enables diagnosis of diverse, ultra-rare monogenic presentations. #GenomicAnalysis #GeneticDiagnosis #UltraRareDisease @natcomms.nature.com 🧬
www.nature.com/articles/s41...

Hello! 👋 I'm looking to connect with other people who have chronic illnesses! #letsbefriends #disabled
#autoimmunedisease #pots #eds #stevensjohnsonsyndrome #ultrararedisease #sunsensitivity #vampire #unknowndisease #chronicillness