RARE X A Research Programme of Global Genes

RARE-X is the technology platform powering the MED13L Data Collection Program. It is part of Global Genes, an organisation dedicated to advancing research for rare diseases. RARE-X removes the barriers typically associated with rare disease data collection by providing a secure, easy-to-use platform that enables families to contribute their health information. By allowing families to share their data, RARE-X helps connect researchers and biopharma companies working on new treatments, accelerating the entire process of discovering new drugs and therapies.

RARE-X ensures that families maintain control over their data, while also making it easier for researchers to access the information they need to make breakthroughs. Participating in RARE-X is a powerful way to help change the future for those living with rare diseases like MED13L syndrome.

At MED13L UK, we are committed to supporting families affected by MED13L syndrome and advancing research that could lead to new treatments. One of the key ways we can all contribute is by participating in the MED13L Data Collection Program, a global initiative aimed at accelerating medical research for rare diseases like MED13L syndrome.

By joining the program, patients, caregivers, and families can share important health information with researchers, helping to unlock critical insights that can speed up the development of new treatments, drugs, and therapies. Your participation helps create a comprehensive dataset that will drive future discoveries and potentially life-changing therapies.

• No clinic visits required – The program is entirely online, and there is no cost to you.
   
• You control your data – As a participant, you decide who has access to your child’s health information. This ensures that your privacy and consent are always respected.
   
• Easy participation – Enrolling in the program takes minimal time, and you can return at your convenience to update your information.
   

Once you join, with your consent, your de-identified data can be accessed by researchers, clinicians, and pharmaceutical companies working to develop new therapies for MED13L syndrome. This data will be used to inform research and support clinical trials that could lead to faster treatment options.

Your participation helps:

• Track disease progression – Researchers can learn how MED13L syndrome changes over time.
   
• Improve clinical trials – Better data leads to more effective trials, reducing the time needed to test new medicines.
   
• Speed up new treatments – The program accelerates the development and availability of new therapies.
   
• Support faster access to therapeutics – Data from participants can even be used as a placebo in trials, reducing the time it takes to develop treatments.

By becoming a part of the MED13L Data Collection Program through RARE-X, you help accelerate research and contribute to the development of new therapies for MED13L syndrome. Join us today, and together, we can unlock the future for those affected by this rare condition.