Your Data Can Help Us Find a Cure

One obstacle in developing drugs and new treatments for rare diseases is a lack of patient data. Only 5% of rare diseases have FDA-approved therapies, and we want ZTTK Syndrome to be one of them! But without valid and reliable data, that won't happen. 

The ZTTK community is partnering with Citizen Health and Rare-X to collect patient data to create natural history studies and help accelerate research, therapy and drug development.

By contributing your data to these studies, you are helping us build the best scientific tools that researchers, clinicians, and biotech partners can use as we progress toward finding a cure. Engaging in research studies is a crucial means of expanding our understanding of ZTTK Syndrome and expediting the development of future treatments and therapies.

Ready to participate in research?

Step 1: Join the ZTTK SON-Shine Foundation’s contact registry so we can contact you on research opportunities. Click here. (~2 minutes)

Step 2: If you live in the US, participate in the Citizen Health to contribute to our ZTTK research database (~5 minutes)

Step 3: For both US and international families, participate in the Rare-X Data Collection Program (~20 minutes initially)

Why is data sharing important?

In order to improve care and develop treatments specific for ZTTK Syndrome, a better understanding of the ZTTK is needed. One way is through collecting information in natural history studies. A natural history study is a type of research study that examines how a disease naturally progresses over time. This can be accomplished in many different ways including (but aren’t limited to) questionnaires and surveys collected over time or reviewing past medical records for trends seen among many patients with the same disorder. Specifically

Inform researchers how ZTTK syndrome change over time

• Enable better data to design and use in clinical trials

• Provide patients the opportunity to participate in clinical trials

• Reduce the time it takes to study new medicines

• Speed up the time to get treatments to patients

• Enable the use of data as a placebo (instead of actual patients) in a clinical trial

There is no cost to participate in Citizen Health or Rare-X data collection programs.

Read more about how a natural history study powered by Citizen Health was used to support an Investigative New Drug (IND) filing with the U.S. Food and Drug Administration (FDA) here

How can I share my or my child’s data?

ZTTK SON-Shine Foundation has partnered with several organizations with the goal to collect natural history information for individuals identified with ZTTK variants. Sharing data and participating in research studies is an important method for increasing what we know about ZTTK patients and accelerating development of future treatments and therapies.

Below is information on all the natural history studies as well as how they complement each other and work together. You can help us speed research efforts right away by building a readily-available pool of data on ZTTK.

Which data collection partner should I sign up with?

If you live in the US, participate in both Citizen Health and Rare-X because they complement each other and work together. It takes 5 minutes to participate in Citizen Health. For Rare-X, you can enroll and fill out surveys over time across sessions.

If you live outside of the US, participate in Rare-X. Citizen is focused on US recruitment to the platform at this time. They are actively reviewing ways to better support international patients in the future and will share updates as we are ready to broaden our availability.