COMBINEDBrain

This report from the National Academy of Sciences came out today. Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. The United States Congress called on the U.S. Food and Drug Administration (FDA) to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union. The resulting report provides recommendations for enhancing and promoting rare disease drug development by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between FDA and the European Medicines Agency. Tagging Global Genes, National Organization for Rare Disorders, and COMBINEDBrain. https://lnkd.in/ezeJRNf9

Attention all attorneys!!! As a #RareDisease advocate and #lawyer, I know first-hand the legal challenges that patient-led rare disease organizations can face as they work to drive forward research towards treatments and cures for their communities. And I also know that there is no more inspiring community of people than the rare disease community. Their grit, determination, and commitment to helping others even when facing tremendous challenge themselves, is unparalleled and deeply inspiring. Today, on Rare Disease Day, #WeTheAction, in partnership with the Chan Zuckerberg Initiative's #RareAsOne Project, launched a pro bono legal support program to support patient-led organizations in the Rare As One Network. I guarantee, without a doubt, your life will be changed for the better in supporting this life-changing work. These organizations, working to move research forward and save lives, need your help. Sign up for one of the listed pro bono projects today! https://lnkd.in/gYF4desA Anna Chu #RareAsOne #raredisease #research #patients #probono #legalsupport

It is Rare Disease Week but I am not participating in DC because Louie Bichell and I are “living the dream”, getting his disease-altering clinical trial treatment…here’s to all the movers and shakers who are making things better for people with rare diseases. Angelman Syndrome Foundation. Foundation for Angelman Syndrome Therapeutics COMBINEDBrain EveryLife Foundation for Rare Diseases National Organization for Rare Disorders, Inc. (NORD) Global Genes Every Cure n-Lorem Foundation.

I am always getting questions from families and one of them is; "What is COMBINEDbrain?" "Should we join CB?" "Who qualifies?" "How is it different from what we are already doing?" The brilliant and totally chill Terry Jo Bichell  explains it all here in this detailed episode. Episode Highlights🧠 Terry's transition from a nurse and midwife to a leader in molecular neuroscience. The birth of COMBINEDBrain, aimed at accelerating research and treatment for rare disorders. An inside look at COMBINEDBrain's impactful services, including a biorepository with over 900 individuals, a free registry for disorder members, and Project FIND-OUT for early diagnosis. The innovative COMBINEDBrain Roadshow, bringing hope and support directly to families across the country. Don't miss this episode of Once Upon a Gene, where passion meets purpose. Tune in to be inspired by COMBINEDBrain's mission to make a difference in the world of rare diseases. 💖🎙