RAREkey - a different kind of patient registry
Putting patients at the centre of therapy development
RAREkey is a free patient registry connecting people with ultra-rare genetic diseases to the researchers and therapy developers working on new treatments. By registering, patients can make themselves known and be considered for relevant clinical trials and new treatment opportunities.
RAREkey also gives patients and families a voice in what comes next. Their priorities, experiences and practical needs can help inform which therapies are developed, what they aim to achieve and how clinical trials are designed.
Why join RAREkey?
For ultra-rare diseases, patients are often scattered across countries and healthcare systems. Therapy developers may not know where treatments are needed or what matters most to the people living with these diseases.
RAREkey gives patients and families a way to make their need for treatment visible and an opportunity to help shape future therapies.
01 – Make your need for treatment visible
Help therapy developers understand where new treatments are needed and whether developing one may be possible.
02 — Help define what a treatment should achieve
Have a voice in what matters most to patients and which outcomes future treatments and clinical trials should focus on.
03 — Inform the design of accessible clinical trials
Share what trial participation would involve for you, giving developers valuable input to consider when designing studies.
Who can register?
RAREkey is free and open to people worldwide living with ultra-rare genetic diseases, regardless of age.
Registrations can be submitted by:
- Patients themselves
- Parents registering on behalf of their child
- Legal guardians authorised to register on behalf of a patient
Required information
Registration is simple and requires only a few details. You do not need to provide a full medical history.
Documents can be submitted in any language.
- Your name and contact details
- Basic patient information, including age and location
- A document identifying the affected gene and specific genetic variant (mutation)
What happens after registration?
Every registration is reviewed by WinQure’s scientific team to assess whether an experimental therapy could potentially be developed. If a relevant opportunity arises, we may contact you to discuss it and hear what matters most to you.
01 – Secure registration
Your information is securely stored in RAREkey. Your genetic documents are reviewed by WinQure and are not publicly accessible.
02 – Scientific review
Our scientific team reviews the genetic diagnosis and assesses whether an experimental therapy could potentially be developed.
03 – Patient input
If a relevant opportunity arises, we may invite you to share your priorities and experiences to help inform therapy development and clinical trial design.
Registration is free and does not guarantee that a therapy will be developed, that you will be eligible for treatment or that you will be offered a place in a clinical trial.
NO PATIENT IS TOO RARE TO MATTER
Make your need for treatment visible. Help shape future therapies.
For too many people living with ultra-rare genetic diseases, the need for treatment remains unseen. RAREkey gives patients and families a way to make that need known and an opportunity to have their voices heard in future therapy development.