KIF1A.ORG

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 impact@kif1a.org kif1a.org/2025-kand-conf…

*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

@StartGenetic is leading an important movement to educated the public about starting with genetic testing if concerning symptoms or developmental delay is detected. Genetic testing early in the process leads to earlier diagnosis, earlier intervention, and better outcomes. Our community knows the benefits of a genetic diagnosis, and we need to help spread the word and encourage others to #startgenetic Read our blog post for more details. kif1a.org/blog/genetic-t…

@StartGenetic Thank you @StartGenetic for creating this movement and inviting other advocacy groups like ours to jump on the bandwagon. This is so IMPORTANT! We must help spread the word and lead others to start with genetic testing sooner!

🧬 Hi, we're Start Genetic, a movement created by parents, for parents to help families understand genetic testing & empower them to discuss it with their child’s pediatrician. ➡️ Tools & resources: startgenetic.org 📰 News: prnewswire.com/news-releases/… #startgenetic

@StartGenetic is leading a critical movement to educate the public about starting with genetic testing at the early signs of developmental delay or concerning symptoms. Our community knows the importance and life changing diagnosis that can come from genetic testing and we want to encourage everyone to support this campaign, share their posts, and spread the word about the first Genetic Testing Day, July 25th. You can also read our blog post for more details. kif1a.org/blog/genetic-t…

rarediseases.org/driving-policy… 💥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/s4143…

🌟 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//ap… @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

@lajollalabs Thank you @lajollalabs for your dedication and support to our patients and community!

Today is KIF1A and Superhero Day! Catch up on the Revealing Rare Genes article that explores KIF1A-Associated Neurological Disorder (KAND) genome.bio/post/personali… @KIF1A #KIF1A #KAND #SuperheroDay #KIF1ADay #raredisease #RevealingRareGenes #lajollalabs #LJL #KIF1Aawareness

🧬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/press-re…

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.us/campaign/11029…

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/campai… youtu.be/a7zoyFIfOQQ?fe…

✨ 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…

✨ 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…

We're kicking off Giving Tuesday for KIF1A.ORG! auctria.events/KIF1A2024Givin… 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

@n_lorem is sharing their scientific advancements in ASO therapy for nano-rare patients. These are modern day MIRACLES! It is truly shocking and amazing what effects patients are seeing with this treatment. Humbled. Blessed. Grateful. n-Lorem. nlorem.org/nano-rare-pati…