When:

July 25-26, 2025

Where:

Wyndham Boston Beacon Hill Hotel

Boston, Massachusetts, USA

Why:

Our bi-annual conference is a beacon of hope for the KAND community. It’s an opportunity to meet with fellow patients and families, engage with leading clinicians and researchers, and build relationships with our biotech partners. Together, we can make a difference.

  • Patients and Families: Connect with others who understand your journey. Gain insights, share experiences, and find support.
  • Clinicians and Researchers: Stay at the forefront of KAND research. Share your latest findings and learn from your peers.
  • Biotech Partners: Understand the needs of our community. Collaborate with us to drive research and development.

Agenda

Meet our Speakers

Dominique Lessard, PhD
Executive Director
Dylan Verden, PhD
Chief Science Officer

Luke Rosen, MS
Co-Founder
Angie Fuller, BSN
Chair
Pat Sarmiere headshot
Patrick Sarmiere, PhD
Translational Science
michelle tao headshot
Michelle Tao, MS, BS
Vice Chair and Secretary
Maria Barros
International Patient Representative
Tamara Agajanov, MD
Clinical Development

Wendy Chung, MD, PhD is a clinical and molecular geneticist and the Chief of the Department of Pediatrics at Boston Children’s Hospital and Harvard Medical School. Dr. Chung has been a champion for KAND since KIF1A.ORG’s founding, and has driven key projects including the Natural History Study and the development of ASO therapies.

Mary Whitman, MD, PhD is an Assistant Professor of Ophthalmology at Boston Children’s Hospital and Harvard Medical School specializing in pediatric ophthalmology and strabismus.  She is one of the few practicing pediatric ophthalmologists who also runs a basic science laboratory; in her lab she studies development of the oculomotor system and genetics of eye movement disorders. She has a special interest in disorders related to kinesins and tubulins and has been performing eye exams as part of the KOALA study of KIF1A related disorder.

Dr. Ebrahimi-Fakhari is a Neurologist and Neuroscientist at Boston Children’s Hospital and Harvard Medical School. He serves as the Director of the Movement Disorders Program and the Pediatric Movement Disorders and Neurogenetics Fellowship at Boston Children’s Hospital. As a physician-scientist dedicated to patient-centered translational research, his goal is to facilitate clinical trial readiness and to develop molecular therapies for genetic movement disorders. Work from this research program has been published in over 130 peer-reviewed publications. His team is supporting the KOALA study and cares for an increasing number of children and young adults with KIF1A-related disorder.

Lottie Morison is a speech pathologist and PhD candidate at the Murdoch Children’s Research Institute in the CRE Translational Centre for Speech Disorders, and The University of Melbourne. Lottie’s research focuses on speech and language in rare genetic conditions, including KIF1A-associated neurological disorder. Lottie’s work aims to improve communication outcomes and quality of life for individuals with KAND and their families. In 2025 Lottie published the first KAND-specific communication study with participation from over 40 families across the world.

Jayne Aiken, PhD, comes from an interdisciplinary background spanning engineering, cell biology, and neuroscience. As a postdoc at the University of Pennsylvania, Jayne has studied how different KIF1A mutations impact the protein’s function, providing key insights into KAND heterogeneity. She was also a key consultant in the selection of KIF1A variants represented in genetically engineered iPSC lines designed by Jackson Laboratory.

Dr. Simran Kaur is an early career researcher and team leader at MCRI and University of Melbourne. Along with her team, Dr. Kaur is leading Australia’s first KIF1A-Associated Neurological Disorder research program in collaboration with national and international collaborators including KIF1A.ORG and KIF1A.AU to accelerate research into the condition. Her group is advancing several key areas, including a compound screen of FDA-approved molecules, development of a KIF1A mini-gene, and incorporating life experiences of KAND families into the scientific literature.

Dr. Laurence Mignon is the executive director of clinical development at n-Lorem Foundation. Laurence has more than 20 years of experience as a seasoned academic researcher and clinical developer with a passion for rare diseases. In her role, Laurence guided n-Lorem’s efforts to create safe RNA-based therapies for nano-rare patients, including several individuals with KIF1A mutations.

Markus Terrey, PhD, is a Study Director in the Rare Disease Translational Center at JAX, where he researches neurodevelopmental and neurodegenerative disorders. The Lutz lab genetically engineers mice to model rare diseases, and has gone above and beyond in developing a suite of tools for KAND research, including mouse models and cell lines available for the KIF1A Research Network.

Parag Gad, PhD, is a co-founder of SpineX, a biotechnology company developing non-invasive neuromodulation to improve movement outcomes in people with spinal cord injury and diseases like cerebral palsy. SpineX’s Spinal Cord Innovation in Pediatrics (SCiP) device recently received Breakthrough Device designation from the FDA.

2023 KAND Conference

  • Patient and Caregiver Stories
  • What matters most?
  • Weighing benefit and risk in exploring new therapies
  • Rare disease advocacy for KAND
  • Growing and supporting the International KIF1A Community

Registration

***In-person registration closing JUNE 15th!

Register now for the KAND Conference to join in person or virtually online—choose the option that works best for you!

Cost: Hosting a conference is a significant undertaking, and the costs can quickly add up—from venue rentals and technology to catering and materials. Despite these expenses, we believe that financial barriers should not prevent anyone from attending. To make our conference accessible to all, we are actively fundraising to cover the event’s costs. If you are in a position to contribute toward the per-person cost, your support would make a big difference. The registration form includes a variety of payment options so you can choose what works best for you. Thank you for helping us bring this important event to life!

Hotel Rooms

***Discounted Rate ends JUNE 21st!

Our event will take place at Wyndham Beacon Hill Hotel in Boston, MA., USA. We have discounted rooms rates for attendees. You have to use this link to receive our discounted rate.

*Some accessible rooms types are booked up at the Wyndham. We recommend staying as close as possible to the Wyndham, however we don’t have another hotel with discounted rates to recommend at this time. A KAND family suggested accessible rooms nearby at the Homewood Suites by Hilton, which include a kitchenette.

Research Participation: KOALA Study

***Enrollment closes May 31st.

Chung Lab at Boston Children’s Hospital will be offering KOALA study participation to our KIF1A patients prior to the conference from July 22-24, 2025. These assessments will take place both at the Boston Children’s Hospital campus and the Wyndham Beacon Hill Hotel in Boston, MA, USA where our conference will be held.

Everyone interested in KOALA participation needs to email ASCENDstudy@childrens.harvard.edu with the subject line: KOALA at the conference and let the team know when you plan to arrive in Boston. Please express your interest in participation early, so the research coordinators have all the information necessary to arrange assessments times, and get everyone the details they are looking for.

EMAIL ASCENDstudy@childrens.harvard.edu

Conference Tshirts

Order your conference t-shirts today! We will have limited sizes and amounts on hand during the actual conference so please plan ahead and order your swag early!

Welcome Social

Everyone is welcome to join us Thursday evening from 7-8:30pm on the top floor of the hotel!

Stay tuned to this page for updates. We can’t wait to see you at the conference!

Together, we are stronger. Together, we can find a treatment and cure for KAND.

2023 KAND Conference
2019 Conference