KIF1A.ORG Newsletter – April 2025
Welcome Our New Executive Director, Dominique Lessard, Ph.D.!
I’m thrilled to share exciting news about the leadership at KIF1A.ORG. Beginning April 14th, Dominique Lessard will officially step into the role of Executive Director, replacing myself, Angie Fuller. Dominique has been a dedicated force within our organization as our previous Chief Science Officer, where she worked tirelessly to bridge scientific research and community impact. Her deep commitment to the KAND community and her scientific expertise make her uniquely suited to guide KIF1A.ORG into its next chapter. Read below for more information about Dom’s previous work experience and please join us in welcoming her back to KIF1A.ORG and her new role!
Dominique “Dom” Lessard, Ph.D.
Executive Director, KIF1A.ORGDominique@kif1a.orgDominique entered the world of KIF1A in 2015 during her time as a doctoral scientist at the University of Vermont, where she characterized how mutations and modifications of the KIF1A protein impact KIF1A function in neurological diseases/disorders. During this time, Dominique connected with KIF1A.ORG and was first introduced to the relentless KAND community. For years Dominique served as a volunteer for KIF1A.ORG with focus on lay-level scientific literacy and communication, aimed at educating and powering the KAND patients, families, and advocates.
In 2020, Dominique joined KIF1A.ORG staff where she served as Chief Science Officer for over three years, with a focus on creating long-term relationships with cross-disciplinary stakeholders. Tasked with leading KIF1A.ORG therapeutic development during this time, Dominique and the KIF1A.ORG team fostered 20+ new projects focused around KIF1A research, established and grew the KIF1A Research Network, and built patient-focused research programs to positively impact the KAND community.
Most recently, Dominique has worked in the rare disease biotech sector, with emphasis on operational oversight and vision, investor relations and financial growth, and program/project management.
For the past 10+ years, KIF1A.ORG and the KAND community have shaped Dominique’s personal and professional life in a multitude of meaningful ways and forms: as an academic researcher, an organization volunteer and employee, a KAND and rare disease advocate, an industry representative and Key Opinion Leader (KOL), and more. Throughout this past decade, the KAND community has remained at the center of Dominique’s personal “why”, focused on empowering patients to have agency over their own treatment and cures while meaningfully weaving the patient voice throughout all elements of scientific discovery. As an equity-driven business strategist and scientist, Dominique is eager to bring the culmination of her skills and experiences back to KIF1A.ORG while wearing a new hat and on a mission to make the next decade even more impactful than the last.
KIF1A Day is April 28!
💚 Join Us for KIF1A Day and Help Our Families Fly!
KIF1A Day is coming up on April 28, and this year we’re celebrating with a powerful campaign to support our community: the Help Them Fly Fundraiser! 🦸♂️✈️
We’re raising funds to help cover travel expenses for families affected by KIF1A-Associated Neurological Disorder (KAND) to attend the 2025 KAND Family & Scientific Engagement Conference in Boston. This life-changing event brings together families, scientists, and clinicians to connect, collaborate, and accelerate research that brings us closer to treatments and a cure.
Here’s how you can help:
💚 Make a donation to support travel and participation for families
🦸♀️ Create a Superhero Page to share patient and families stories, raise awareness, and fundraise for the cause
Every dollar brings us closer to our goal—and helps make it possible for families to take part in critical research and connect with others facing the KAND journey.
Let’s lift up our heroes and Help Them Fly this KIF1A Day!
**If you are participating in the KOALA study at the conference, and would like to request travel assistance, please email us at Events@kif1a.org!
KAND Family & Scientific Conference: Register Now!
Don’t miss the 2025 KAND Family & Scientific Conference—a one-of-a-kind opportunity to connect with other families, researchers, and clinicians who are dedicated to advancing understanding and treatment of KAND.
Register Today and be part of this transformative gathering!
Participate in the KOALA Study at the Conference
Thinking about joining the KOALA Study? The timing couldn’t be better! 🐨✈️
Participating in KOALA just before the KAND Conference is the perfect way to knock out two big opportunities in one trip to Boston. You can contribute to vital research and connect with the KIF1A community—all in the same visit!
We still have about 10 spots left for KOALA visits before the conference, so if you’re already planning to attend, consider coming a little early to be part of this important study.
Help us move research forward—one visit, one trip, two huge impacts.
Extraordinary Gift from Jersey, UK
We are incredibly grateful to announce a generous donation from a private trust in Jersey, UK, which will significantly strengthen our research initiatives. This gift will help us accelerate scientific advancement and bring us closer to meaningful treatments for KAND.
A heartfelt thank you to Madlen Wollersheim from Germany for recommending KIF1A.ORG to the trustees and helping make this impactful support possible.
Neuromodulation: SpineX
KIF1A.ORG is excited to announce the launch of a groundbreaking pilot project in partnership with SpineX Inc., exploring the potential of neuromodulation therapy for children living with KIF1A-Associated Neurological Disorder (KAND). This marks a promising new chapter in our efforts to explore innovative therapies that could improve quality of life for KAND patients.
As of this month, two children with KIF1A have begun participating in the first-ever neuromodulation pilot study for our community. This initial two-patient study will help gather critical data over the coming months to evaluate the feasibility and effects of this therapy in individuals affected by KAND.
What is Neuromodulation?
Neuromodulation involves delivering gentle electrical stimulation to the spinal cord or nervous system to improve communication between the brain and body. It does not involve surgery or medication, and is designed to be a non-invasive, safe way to help “retrain” the nervous system.
In prior studies with children who have cerebral palsy, neuromodulation has led to improved motor function, increased balance, and better posture and gait — even in children with severe mobility challenges. These promising results provide a foundation for testing whether similar benefits can be seen in children with KAND, many of whom experience motor dysfunction due to degeneration of the central and peripheral nervous system.
Why This Matters for KAND
Motor symptoms are among the most challenging and life-altering features of KAND. By targeting the spinal cord’s communication networks, neuromodulation offers a potential way to enhance movement, strength, and coordination — and perhaps open the door to improved daily function and independence.
As this pilot project progresses, we look forward to sharing updates and learnings with the KIF1A community. We’re deeply grateful to the families participating in this important study, as well as to our collaborators at SpineX, who bring expertise and a shared commitment to pushing boundaries for children with complex neurological conditions.
Stay tuned — and thank you for powering our mission to accelerate research and bring hope to every family affected by KAND.
Wearable Digital Data Collection: BioSensics
KIF1A.ORG is thrilled to announce an exciting new initiative to enhance our natural history study and accelerate progress toward clinical trials: wearable digital data collection in partnership with BioSensics, a leading innovator in digital health monitoring for rare diseases.
As part of our efforts to strengthen the KOALA Study—the most comprehensive natural history study of KIF1A-Associated Neurological Disorder (KAND)—we are now piloting cutting-edge wearable devices to gather continuous, real-world data from patients at home. These devices are designed to capture critical metrics such as:
- Movement
- Gait
- Balance
…and more.
How It Works
Patients are first evaluated in-person during KOALA study visits at Boston Children’s Hospital. After their visit, they are sent home with these lightweight, non-invasive wearable monitors provided by BioSensics. The devices passively record vital data over time, painting a fuller picture of each patient’s day-to-day experience—something a clinic visit alone can’t always capture.
We are currently piloting this technology with a select group of families during regular KOALA visits. Our goal is to expand this offering to all KOALA participants during the upcoming KAND Conference, giving every family the opportunity to contribute valuable at-home data to our growing research efforts.
Why It Matters
These additional data points give researchers a more robust, dynamic view of how KAND affects individuals in real life—outside the clinic. This is especially important for rare diseases, where every data point counts. By building a richer dataset, we not only improve our understanding of the progression and variability of KAND, but also make significant strides toward being clinical trial ready when a treatment becomes available.
Thanks to our collaboration with BioSensics and the dedication of the KAND community, we’re unlocking new possibilities in rare disease research—and bringing hope one step closer.
Stay tuned for more updates as we continue to scale this effort and push the boundaries of what’s possible in KIF1A science.
Volunteer Spotlight: David Ruff
David is a passionate volunteer with KIF1A.ORG who lives in North Jersey and commutes to New York City for work. He holds a degree in Biology from The College of New Jersey, with minors in Chemistry and Psychology, and is currently finishing his master’s thesis in Biotechnology at Columbia University. David has a strong background in scientific research, having worked at a neurology lab at Columbia University Medical Center studying brain dysfunctions related to movement disorders. His passion for helping those affected by rare genetic conditions drives his career goals. Outside of work and volunteering, David enjoys hiking and camping, and he is an advocate for New Jersey’s natural beauty. He was inspired to join KIF1A.ORG after hearing Luke Rosen’s seminar during a Biotech class at Columbia, which reminded him of his family’s experience with Rett syndrome. You may have met David at the 2023 KAND Conference helping with the Audio Visual systems, ensuring we could hear, see, and record all presentations. Thank you David for your continued support and dedication to improving the lives of our patients!
To learn more about how you can get involved, explore our current volunteer opportunities here!
We can’t wait to connect with you at our upcoming virtual and in-person events! Take a look at what’s coming up.
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Apr 28
KIF1A Day
💚 April 28, 2025: KIF1A Day Every April 28th, we come together to honor our community on KIF1A Day—a day to celebrate our incredible patients and raise…
Monday, April 28, 2025 12:01 am – 11:59 pm Globally -
May 14
2nd Quarter Community Call, KIF1A.ORG
Join us each quarter to dive into current community events, exciting research opportunities, scientific updates, and more! Your participation is essential, and we truly value…
Wednesday, May 14, 2025 1:00 pm – 2:30 pm Eastern Standard Time
Online via Zoom -
May 15
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,…
Thursday, May 15, 2025 12:30 pm – 2:00 pm Eastern Time Zone
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 -
Aug 28
3rd Quarter Community Call, KIF1A.ORG
Join us each quarter to dive into current community events, exciting research opportunities, scientific updates, and more! Your participation is essential, and we truly value…
Thursday, August 28, 2025 12:30 pm – 2:00 pm Eastern Time Zone
Online via Zoom
📢 ACTION ITEMS Reminder:
- Register for the conference (in-person or virtual attendance available)
- Register to participate in KOALA before the conference (10 spots left!)
- Create a KAND patient Superhero Page, if you haven’t already
- Share your superhero story across social media in the days surrounding KIF1A Day, with a donate link to our KIF1A Day page and progress bar
- Donate to the “Help Them Fly” Fundraiser!
Thank you for being a part of this extraordinary community. Your support, passion, and participation are what drive this mission forward. We’re in this together—and together, we’re unstoppable.
With hope,
The KIF1A.ORG Team