September was packed with informational meetings, grant submissions, global connections with scientists, impact reports, awards, and outstanding community fundraising achievements! Keep reading to discover these amazing milestones and learn how you can get involved to make an IMPACT too!
UPCOMING MEETINGS
*Times are Eastern Time Zone.
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Jan 22
Newly Diagnosed Meetup
These gatherings are open to anyone that is new to our community or any KAND families interested in learning more about our organization’s mission, staff,…
Wednesday, January 22, 2025 1:00 pm – 2:30 pm Online via Zoom -
Jul 22 – 24
KOALA Study prior to KAND Conference
The KOALA study will be available to an estimated 40 KAND patients in the days leading up to the conference, on July 22, 23, and…
Tuesday, July 22, 2025 – Thursday, July 24, 2025 In-person
Boston, Massachusetts, USA -
Jul 25 – 26
2025 KAND Family & Scientific Engagement Conference
We’re excited to announce the official dates for our 2025 KAND Conference! Mark your calendars for July 25-26, 2025, and plan to join us in…
Friday, July 25, 2025 – Saturday, July 26, 2025 In-person and Virtual
MCRI Impact Report
In 2020, we provided MCRI and Dr. Kaur with $125,000 to initiate a small molecule drug screening project focused on KIF1A-associated neurological disorder (KAND). Since then, Dr. Kaur has taken that initial investment and transformed it into a remarkable success story, raising over $1 million to expand and accelerate a comprehensive KIF1A program in Australia. Her leadership and vision have resulted in the establishment of crucial research initiatives, including a vision study and a speech study, alongside the development of gene therapy approaches and biomarkers specifically for KAND.
Dr. Kaur’s dedication has also led to the creation of a biobank, which will serve as a vital resource for future research, and she has made significant strides in engaging with both the patient community and regulatory bodies to explore how to bring ASO treatments to Australia.
We are deeply grateful for Dr. Kaur’s incredible commitment and tireless efforts to push the boundaries of KAND research. Her work has had a profound impact on our community, and we cannot thank her enough for her vision, passion, and unwavering dedication to improving the lives of those affected by KAND.
Congratulations Dr. Chung!
Congratulations to Dr. Chung and her amazing team on the Global Genes Rare Champion of Health Equity 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!
Are your ASCEND surveys complete?
We would like to remind all participants in the ASCEND study that some of you may have unfinished surveys that you may not be aware of. It’s important to ensure that all surveys are completed for the study to be as thorough and impactful as possible. We kindly ask that you check your survey link and survey queue to confirm that all required surveys have been completed and that you are up to date. Your participation and diligence are critical to the success of the study, and we greatly appreciate your time and effort in helping us gather this valuable data. This video describes how you can check for unfinished surveys. Email Angie at Angie@kif1a.org with any questions.
3rd Quarter Community Call Video
On September 18th, we hosted a productive and informative community call, covering three recent KAND publications: the natural history study, vision findings, and ASO therapy as well as NeuCyte compounds and how we are thinking about those moving forward.
Dr. Dylan Verden’s summaries during the call provided clear insights into the key takeaways. You can find the recordings under the Community section of our website, Private Family Resources, under Community Calls. If you need assistance, please reach out to Angie at Angie@kif1a.org.
Volunteers needed to join the team!
Volunteers are the heart of our organization, and we couldn’t thrive without them!
We are currently seeking many positions. The time commitment of most volunteer roles is approximately 5 hours per week, with the possibility of additional hours as needed.
If you’re interested in learning more about how you can help, please look at our Volunteer webpage and email Angie at Angie@kif1a.org!
Volunteer Spotlight
We are thrilled to spotlight Bonny Briney, a dedicated volunteer and mother of KAND patient Maddox. Living in Southern California with her husband Doug and their younger son Landon, Bonny is always on the move with two energetic kids. Despite her busy life, she has worn many hats in our organization, from fundraising and creating social media posts to designing t-shirts and helping us secure a hotel for our upcoming KAND conference. Bonny’s tireless dedication and selflessness have had a tremendous impact on our community, and we are deeply grateful for her contributions!
Research enrollment numbers
We added 7 additional patients and families to our Natural History Study last month!
We are deeply grateful for your participation and your invaluable contributions to advancing our research efforts.
We know that living with a rare disease can be an overwhelming and isolating journey. The daily challenges you face are not just physical but emotional, and the uncertainty of the future can be daunting. But there is a powerful way you can help change this—by becoming a part of our natural history study.
By enrolling in this online research study, you are not just a participant; you are a crucial partner in the fight to understand and ultimately find treatments for KAND. Your unique variant, experience, and data are invaluable to researchers working tirelessly to uncover the mysteries of this condition. No one else can represent you in this research, and we don’t want to leave anyone behind and unaccounted for.
If you or your loved one has a KIF1A variant, please consider enrolling in our natural history study. Be a part of this critical mission to advance research, bring awareness, and, most importantly, make a difference. To enroll, fill out this interest form and researchers at Boston Children’s Hospital will email you back with next steps. *Add this email to your contacts so emails don’t get lost in spam folders: ASCENDstudy@childrens.harvard.edu
TAP Fundraiser~ WE DID IT!
We are thrilled to announce that, thanks to the incredible generosity and dedication of our community, we have successfully reached our fundraising goal of $70,000 to begin testing compounds at NeuCyte! This achievement marks a significant milestone in our journey toward finding treatments for KIF1A-associated neurological disorder (KAND). We couldn’t have done this without the tireless support of every donor and advocate. Together, we are creating hope and paving the way for life-changing progress! Thank you for being part of this incredible accomplishment.
Thank you Valerie, Bryn, and Bonny!
We want to extend our deepest gratitude to three incredible KAND moms and their families, who recently raised an extraordinary $37,000 combined for KIF1A.ORG!!! Your dedication, hard work, and passion for making a difference have had a profound impact on our community. The funds you’ve raised will help drive critical research and bring us closer to breakthroughs that will change lives. We are so grateful for your support and your commitment to the KIF1A cause.
Thank you for being such inspiring advocates and for showing what the power of community can achieve!
Thank you!
As always, we extend our heartfelt gratitude for staying informed and engaged with KIF1A.ORG. Your ongoing participation and commitment to our mission are truly invaluable, and the impact you have on advancing our collective goals cannot be overstated. Together, we are making a profound difference in the lives of those affected by KIF1A-related disorders. Thank you for your continued support, dedication, and partnership in this journey!