Why Should You Participate in Research?

Participating in research can unlock benefits for you today, like access to expert care. You are also investing in your future quality of life, as Kabuki syndrome is a lifelong condition. And you are helping others – those here today and for generations to come.

Your participation is crucial in the patient-centered research we support and that recognizes your voice at each step on the roadmap to treatments.

How You Drive Research to Treatments

As a person with a rare disease or caregiver, your unique experiences tell a story that can blaze a path from the lab to clinical trials. Your participation is your power to help find treatments.

Ensure research priorities match your needs.

By participating in research, you can make your voice heard. Your perspective is invaluable for researchers exploring treatments. From what symptom is targeted to how and when a drug is given, researchers can aim to meet your needs and priorities if you share them during treatment development.

Improve the standard of care for Kabuki syndrome.

Research provides an opportunity for our community to come together and pool our collective knowledge and experiences. By sharing your insights and data, you help cultivate a deeper understanding of Kabuki syndrome. We can create a map of what to expect at every age, and what the best treatments are for people with Kabuki syndrome.

Unlock life-changing treatments.

Your samples, data, and time are necessary catalysts at each stage of drug development. Without your participation, treatment development will stall. We are parents, patients, and clinicians driving research to treatments, and it’s going to take all of us.

"I want to change the research landscape."

Kristin and Mathew, Kabuki syndrome research participants, are driven to help shape the direction of research and to help others. They shared their experiences live at a past research conference and continue to support others interested in participating.

“I participate to learn more about Kabuki syndrome for myself and to inspire others to participate.”Mathew, KSF Research Ambassador

Join Mathew, Kristin, and hundreds of others in participating today. 

Current Opportunities

Patient-centered research is the only path forward.

To take better care of our patients, we ask: What is important to you? What impacts you the most? And how can we approach research to find a better way to answer those questions for you?” -Dr. Brittany Simpson, Kabuki syndrome expert clinician and researcher

Your input is critical as investigators like Dr. Simpson develop and advance patient-centered research toward treatments. We are the bridge between the community and the investigators, amplifying your voices and creating opportunities for you to share and show your priorities at every stage of treatment development.

Patient-centered research is more efficient and reduces burdens for families.

Clinical trials can be designed to better address your needs and to reduce barriers you could experience as a participant. Examples of possible barriers could include travel requirements and associated expenses. Collaborative efforts between researchers, patients, and research-focused organizations like KSF are crucial in the creation of effective, accessible clinical trials.

Dr. Adam Hartman from the NIH further illustrated your critical role in clinical trial design at our 2022 Research Conference.

Watch More About Clinical Trial Design

Ready to Find An Opportunity that Fits Your Family?

Join a study from home or in-person, or explore other ways to help drive research to treatments.