2025 KAND Conference Presentations & Slides Now Available! Conference recordings and presentation materials can now be accessed through our website or YouTube channel. Don’t miss the Highlight Video featured at the top of the page—a chance to relive the inspiring moments of our unforgettable weekend together. For any questions, please contact us at [email protected] kif1a.org/2025-kand-conferen…

*Turn sound on* ✨2025 KAND Conference Highlight Video✨ We’re still buzzing from an incredible weekend together! 💙 Here’s a look at some of the unforgettable moments from the 2025 KAND Conference. Full presentations will be released later today or tomorrow—so check our website to catch every inspiring session from our families, researchers, and clinicians. 🎥✨ kif1a.org

rarediseases.org/driving-pol… 💥Medicaid and Lifesaving Waiver Programs Are Under Threat A new bill backed by Trump proposes massive cuts to Medicaid funding—putting essential care and services for millions of families, including ours, at risk. ✅Take 2 minutes to make your voice heard. This simple tool lets you send a prewritten email to your senators urging them to protect Medicaid. You can also add a few sentences about how Medicaid has impacted your life or the life of people you know and love. 🙋‍♂️Your story matters. Your voice matters. Alone we are rare. Together we are STRONG. 🧬

The first KAND-focused speech and communication study: led by Professor Angela Morgan and Lottie Morison at @MCRI_for_kids, joined by Dr. Wendy Chung, KIF1A.ORG's Dylan Verden, and 44 KAND families! nature.com/articles/s41431-0…

🌟 Today, on KIF1A Day, we celebrate the incredible progress our community is making — and the people who are helping to lead the way. At KIF1A.ORG, we are honored to work alongside a remarkable network of researchers, scientists, and clinicians who are dedicated to advancing our understanding of KIF1A and KIF1A-Associated Neurological Disorder (KAND). Their partnership with our organization and our patient families is accelerating research, deepening knowledge, and moving us closer every day to effective treatments and, ultimately, a cure. Breakthroughs in rare disease research don’t happen in isolation. They are built through collaboration, passion, and relentless pursuit — values that define the extraordinary individuals who stand with our community. But today, we especially celebrate the true heroes of this journey: our patients. Their courage, resilience, and determination inspire every step forward. On KIF1A Day, we honor them — and commit to making sure their voices and needs lead the way. This summer, we will gather for the KAND Family & Scientific Engagement Conference — a powerful opportunity to connect patients, families, scientists, and clinicians in person. To make this event accessible to as many families as possible, we are raising funds to help cover travel expenses for families who otherwise couldn’t attend. 🎉 Join us in celebrating our patients today — and help us bring them together this summer! Your support will help create life-changing moments of connection, learning, and hope. 👉 Donate today and be part of powering our community forward. kif1a.org/april-28th//april-… @WendyKChung @n_lorem @BainBrainLab @jacksonlab @neucyte @MCRI_for_kids @MCRI_SpeechLang @ChanZuckerberg @Coriell_Science @Columbia @BostonChildrens @chrisricupero @EinsteinMed @OvidRx @bioloomics @OnceUponAGene @IAmBiotech @arnegennerich @RareDiseases @EveryLifeOrg @GlobalGenes @bflay @newyorkbio

🧬Today, on KIF1A Day, we want to extend our heartfelt gratitude to @n_lorem for bringing hope to the KIF1A community. n-Lorem’s commitment to developing individualized RNA-based therapies for patients with ultra-rare genetic diseases is truly life-changing. Their innovative work is offering new possibilities — and real hope — to KIF1A patients and families who once had none. By partnering with patients, families, and researchers, n-Lorem is not only advancing science, but also shining a light on what’s possible when compassion and innovation come together. Thank you, n-Lorem, for standing with our community and helping to change the future for those affected by KAND. We are deeply grateful for your partnership and your unwavering dedication. 💚 #KIF1ADay #KAND #nLorem #RareDisease #HopeInAction #GeneTherapy #ThankYou

“We must ensure patients suffering from rare and chronic diseases have access to lifesaving medications for years to come, By incentivizing innovation in breakthrough treatments, the MINI Act is a win for patients, a win for breakthrough scientific research, and a win for common sense, bipartisan solutions,” said Congressman Don Davis. Yesterday, Congressman Davis introduced the bipartisan Maintaining Investments in New Innovation (MINI) Act, legislation ensuring continued access to medical therapies developed with genetically targeted technology (GTT). KIF1A.ORG's Director Angie Fuller is quoted in the legislation, advocating for our patients and GTT innovation! Take a look! dondavis.house.gov/media/pre…

For the 30 million Americans living with one or more rare diseases, steady federal support for robust biomedical research and stability at national public health agencies are essential to advancing lifesaving research and public health initiatives. This weekend's removal of key experts at Health and Human Services Agencies including NIH, FDA, and CDC – paired with recent executive orders on federal funding and public health agencies -- will have a devastating and lasting impact on programs that are vital to thousands of rare disease communities, and our nation as a whole. everylifefoundation.quorum.u…

Our Research Network member, MCRI's Dr. Simran Kaur, leads critical research into KIF1A/KAND, with goals in the short, medium and long-term to provide treatment options and ultimately work towards a cure. Learn more about how this research provides hope for families who are dealing with KIF1A/KAND. This video features Australian patients Zia, Edward and Sophia, who all have a unique journey, and experience, with the disease. We are so grateful for Dr Kaur's dedication and relentless pursuit of treatment and a cure. To donate to her KIF1A/KAND research, head to mcri.edu.au/support/campaign… youtu.be/a7zoyFIfOQQ?feature…

✨ UPDATE! ✨ We are absolutely blown away by the outpouring of support and love for our community! Thanks to your incredible generosity, we’ve raised an amazing $25,916, including matching contributions! Your kindness and dedication are truly inspiring. THANK YOU for standing with us and fueling our mission. Together, we’re making a difference and so close to reaching our $30,000 goal! 💙🎉 kif1a.org/giving-tuesday-202…

✨ Today is Giving Tuesday!✨ Thanks to your incredible generosity across all donation platforms so far, we’ve raised over $13,000, including matching contributions! THANK YOU SO MUCH! 💙🎉 Please continue to share and donate so we can reach our goal of $30,000 and keep our urgent mission alive! kif1a.org/giving-tuesday-202…

We're kicking off Giving Tuesday for KIF1A.ORG! auctria.events/KIF1A2024Givi… Join us as we raise money to keep our urgent mission alive!

Happy Thanksgiving, KAND Family! Today, we want to take a moment to share how truly grateful we are for each of you. Together, we’ve built an incredible support network filled with love, strength, and hope. Through the challenges, you remind us of the power of community and how much we can achieve when we stand together. You are the heart of everything we do, and we are so thankful for the connections we’ve created and the hope we share for a brighter future. Wishing you and your loved ones a joyful and peaceful Thanksgiving! With love, KIF1A.ORG

Congratulations to @WendyKChung and her amazing team on this well-deserved award! No one is more deserving of this recognition. Your relentless pursuit of answers and solutions for our nano-rare community has made a profound impact. We are incredibly grateful for your team's dedication, passion, and tireless commitment to improving the lives of those affected by KAND. Thank you for inspiring us all and continuing to lead the way toward groundbreaking discoveries!

We are incredibly grateful for the partnership and the relentless work being done for KAND at Australia's @MCRI_for_kids ! Their annual report highlights the efforts led by Dr Simran Kaur, a longstanding member of the KIF1A.ORG Research Network and a passionate advocate for KAND. Thank you, MCRI, for sharing your Annual Report and for spotlighting the amazing work you're doing to support KAND families worldwide. With hope, KIF1A.ORG & KIF1A Australia Foundation issuu.com/murdochchildrens/d…

✨Canadian KAND patient, Hendrik, is featured in a news article from Yahoo!Life. Take a look~ ca.style.yahoo.com/it-is-a-n…

✨ Exciting News! ✨ We are thrilled to announce the publication of the **first-ever KAND ASO study**! This groundbreaking research marks a significant milestone in the fight against KAND, and it would not have been possible without the incredible collaboration and dedication of our team and partners. A heartfelt thank you to Dr. Chung and Dr. Bain for leading the charge with unmatched expertise and passion. We also extend our deepest gratitude to n-Lorem for creating and providing the ASO drug that made this trial possible. And most importantly, our utmost respect and appreciation go to the pioneering family and patient who courageously participated in this trial. Your bravery paves the way for future breakthroughs and brings hope to many. Together, we are making history and bringing hope to the KAND community! #KIF1A #KANDResearch #MedicalBreakthrough #Gratitude #HopeForTheFuture @BainBrainLab @n_lorem #wchunglab #KANDASO #ASO Article: nature.com/articles/s41591-0…