We are a leading patient advocacy organization dedicating to advancing research of Leigh syndrome and empowering and supporting affected families worldwide. Our mission is to unite the global Leigh syndrome community to accelerate patient-centered research, treatments, and cures. We fund research and provide comprehensive resources for the community, including a global patient registry for Leigh syndrome, an educational website AboutLeighSyndrome.com, Leigh syndrome healthcare providers directory, annual Leigh syndrome virtual symposium, support resources for patients, and opportunities for free genetic testing, among other initiatives. Our foundation is volunteer run and parent led, 100% of your donations are tax-deductible and will go directly to research dedicated to mitochondrial diseases. You can learn more at our website: https://www.curemito.org/ You can donate at: https://www.curemito.org/ways-to-give You can learn more about Leigh syndrome global patient registry at: https://www.curemito.org/leighsyndromeregistry
Cure Mito Foundation的外部链接
US，Texas，McKinney
Associate Director, Statistical Programming at Alexion | Vice President and patient registry director at Cure Mito Foundation | Rare Disease patient advocate
I'm thrilled to be leading a workshop at this year's World Orphan Drug Congress USA on "Patient Registries: A Practical Approach for Patient Foundations." If you're attending, please reach out—I'd love to connect!
Researchers tested HypoxyStat, a small molecule that mimics the effects of hypoxia by increasing hemoglobin’s oxygen affinity, and found it significantly extended lifespan and improved multiple disease features in a mouse model of Leigh syndrome. Unlike gas-based hypoxia therapies, HypoxyStat offers a practical, orally administered approach with potential for clinical use. Learn more in the new paper by Isha H. Jain et al. published in Cell by Cell Press https://lnkd.in/ejW3-Zhi Isha Jain #Leighsyndrome #mitochondrialdisease
Save the dates! September 16, 2025 - Empower and Inspire: 4th Annual Leigh Syndrome Symposium. September 17, 2025 - Leigh Syndrome Awareness Day. More information to come, stay tuned! #mitochondrialdisease #leighsyndrome
?? Check out our latest newsletter! Read and share to help us make a difference: https://lnkd.in/d8DGGzR3 #CureMito #MitochondrialDisease #LeighSyndrome
Mitochondrial DNA diseases present unique challenges in genetic counseling, influencing options like IVF, prenatal diagnosis, and emerging techniques like gene editing. New paper by Dr. Bj?rn Heindryckx et al. emphasizes the need for personalized approaches and careful consideration of potential benefits and risks. Learn more at: https://lnkd.in/ebri697K Oxford University Press
?? Attention researchers! Explore our new resource featuring a collection of animal models, fibroblasts, and other essential biosamples. Learn more at: https://lnkd.in/eY5Rdkat #LeighSyndrome
?? Introducing The CRID – Your Universal Clinical Research ID! ?? The CRID securely links your medical records to you, enabling your data to seamlessly contribute to essential research. Soon, we'll provide details on how you can use your CRID to connect your registry data with your biospecimens or any other research studies you're participating in. We encourage everyone to learn more at https://lnkd.in/exYvCx3x and to create your CRID at https://thecrid.org/.
Have you seen our new animated video about Leigh syndrome yet? Find it below on our YouTube channel and share! https://lnkd.in/dwKxNhEQ
Mitochondrial diseases are complex and largely untreatable due to their effects on vital organs like the brain and heart, and challenges in mitochondrial DNA engineering. However, human induced pluripotent stem cells (iPSCs) offer new hope. Increasingly used in research, iPSCs help create models to study these diseases and explore treatment options in a way that's specific to the patient and tissue affected. Recent advances in iPSC-derived models, including both 2D cells and 3D organoids, are paving the way for breakthroughs in treatments and understanding of mitochondrial diseases. Learn more in the new paper by Dr. Alessandro Prigione and Dr. Sonja Heiduschka We are proud to have supported this research along with other organizations https://lnkd.in/eqGhg3bE
Thank you, Marshall family, for your continued support and fight for Zander and so many others. Thank you Indiana Gazette for sharing Zander's story. https://lnkd.in/eiCBg4Ma