Hello, amazing KAND Community!
I know I might sound like a broken record, but we have SO MUCH exciting news and information to share this month! Here’s a sneak peek at what’s in store:
- Upcoming Meetings: Newly Diagnosed Meetup, 3rd Quarter Community Call, & Research Roundtable
- ASO Study Publication: We’re thrilled to announce the groundbreaking publication of our first-ever ASO study—an incredible milestone for our community!
- Sloane’s 3rd ASO Dose Update: Hear the latest on Sloane’s journey and her third ASO dose.
- 2025 Conference Interest Survey: Help shape the future of our community by sharing your interest in the upcoming 2025 conference.
- Volunteer Request: Looking for passionate volunteers to help drive our mission forward!
- Research Enrollment Numbers: Check out the latest stats and see how our research efforts are expanding.
- Research Network Grants: Learn how KIF1A.ORG can help our research network members apply for grants.
- TAP Fundraiser Update: Get the latest scoop on our fundraising efforts and how you can get involved!
- Superhero Stories: Be inspired by the incredible stories from our community’s superheroes.
Let’s dive in and explore all these wonderful updates together!
UPCOMING MEETINGS
*Times are Eastern Time Zone.
-
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
Boston, Massachusetts, USA
ASO therapy: Positive Clinical Outcomes Observed
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!
For a summary of the scientific publication, you can read Dr. Dylan Verden’s blog post and n-Lorem’s press release.
If you would like to read the full article, please email impact@kif1a.org and request the ASO Therapy article.
2nd KAND patient receiving ASO treatment
Meet Sloane. She is the 2nd KAND Pioneer blazing the trail with ASO therapy. Her mom, Megan, regularly updates the community after each dose.
Sloane recently received her 3rd dose and her mom has this to say:
Hello KIF1A Families,
Sloane received her third dose of the ASO on August 1st. We had pre-dose bloodwork, PT evaluation, and research EEG and then post-dose bloodwork, EKG, and research EEG. No substantial motor changes from what we were already seeing with this dose, but we did have one huge milestone this month…Sloane’s first day of school! Right now we are focused on how to maximize what insurance is paying. We will head back to New York in October for Sloane’s fourth dose!
With Hope,
Megan, Sloane, and family
*You can read previous blog posts about Sloane’s journey here!
2025 KAND Conference Interest Survey
Calling all KAND Community Stakeholders! We are reaching out to assess interest in hosting another Family and Scientific Engagement Conference in Boston, July 2025! We kindly ask that everyone completes this 5 minute survey.
*KAND families, please only complete one survey.
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 including a volunteer administrative assistant to support us with tasks such as calendar management, meeting coordination, record keeping, data entry, and correspondence. 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!
Research enrollment numbers
A heartfelt THANK YOU to the 15 KAND patients and families who joined 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
Research Network – Applying for grants? Let us know
We know that getting research funded is a constant challenge, and we’re eager to help! If you are currently applying for any KAND-related grants, please let us know how we can assist:
- Brainstorming and Design
- Integration of Research Network Resources
- Letters of Support
- Significance/Patient Relevance Sections
- Proofreading/Editing Services
External Grant Opportunities:
California Institute for Regenerative Medicine – Discovery and Translation grants
- Description: “The path from stem cell therapy development to life-saving treatments can be challenging, but here at CIRM we have developed a series of funding programs that together are poised to improve the efficiency by which much-needed therapies reach patients in need.” While the primary focus of CIRM grants has been cell therapy, it has also promoted the use of cell-based disease modeling and compound screening, as well as the development of gene-based therapies.
- Eligibility: California-based research groups
TAP Fundraiser
Our community is raising funds to support research on KIF1A mutations and potential therapeutic compounds in collaboration with our partners at NeuCyte. Our goal is to raise $70,000 USD to kickstart this project. We’re almost there, with 70% of the funds raised so far! To learn more, visit our TAP webpage.
Superhero Stories
A heartfelt thank you to the families who generously share their patient journeys through our Superhero Stories. Your courage and resilience inspire us all. We invite you to explore their touching stories and photos, which capture the strength and hope within our community.
If you’re inspired to share your own journey, we would be honored to feature your story. Submit your Superhero Story using this here. Your voice matters, and together, we can continue to uplift and support one another.
