Register with RAREkey

Make your need for treatment visible. Help shape future therapies.

RAREkey is a free patient registry for people living with ultra-rare genetic diseases. By registering, you can make your need for treatment known to WinQure Medicine and help us understand where new therapies are needed. RAREkey also gives patients and families an opportunity to help shape future therapies, including what a treatment should achieve and how clinical trials can be designed around patients' needs. If a relevant therapeutic opportunity arises, we may reach out to discuss what matters most to you, how the disease affects everyday life and what would make participating in a clinical trial possible. WinQure's scientific team reviews the genetic information submitted to assess whether an experimental therapy could potentially be developed. Registration is free and does not constitute an application to a WinQure programme, guarantee eligibility for any future programme or indicate that a treatment is currently available or under development for the patient's disease.

Diseases know no borders. Genetic testing reports and supporting information can be submitted in any language.

Who are you registering for?(Required)

Myself

My child or someone for whom I am the legal guardian

1. Your information

Please provide your own details, even if you are registering on behalf of a patient.

Full name(Required)

First name Last name

Email address(Required)

Enter Email Confirm Email

Phone number(Required)

Country Phone Number

Address(Required)

City or Region Country of Residence

2. About the patient

Please provide some basic information about the person living with the genetic disease.

Patient age at time of submission(Required)

Please enter a number from 0 to 110.

Patient country of residence at time of submission(Required)

3. Upload genetic test results

To assess whether an experimental therapy could potentially be developed, our scientific team needs to know the gene and specific genetic variant (mutation) responsible for the disease. Please upload a genetic test report or other medical document that clearly identifies the gene and variant.

Where can I find my genetic test results?

Your results may have been provided by your treating physician, geneticist, genetic counselor, hospital or genetic testing laboratory. If you do not have a copy, you can request one from the healthcare provider or hospital that arranged your genetic testing. We only need the document or relevant pages showing the gene and specific variant. You do not need to submit your full medical history.

Upload genetic test results(Required)

Accepted file types: pdf, docx, jpg, png, Max. file size: 1 GB.

Documents in any language are welcome.

4. Additional information

Is there anything else you would like us to know?

You may include information about existing research, patient communities or anything else you consider relevant.

You do not need to provide details about symptoms, treatment priorities or your ability to participate in a clinical trial at this stage.

If a relevant therapeutic opportunity arises, we may contact you to discuss these topics directly.

We want patients and families to have a voice in defining what a treatment should achieve and how clinical trials are organised. These discussions will help us understand what matters most to you and how participation can be made as accessible as possible.

5. Registration and consent

RAREkey accepts registrations from patients themselves or from parents and legal guardians who are legally authorised to act on the patient's behalf.

To register with RAREkey, we require your consent to collect, securely store and review the information you provide, including genetic and health information, and to contact you about your registration and relevant therapeutic opportunities.

WinQure Medicine ApS is the data controller responsible for processing personal information submitted through RAREkey.

The legal basis for consent-based processing under the General Data Protection Regulation (GDPR) is Article 6(1)(a), together with Article 9(2)(a) for the processing of genetic and health information.

Consent may be withdrawn at any time by contacting [email protected]. Withdrawal does not affect the lawfulness of processing carried out before withdrawal.

6. External therapeutic opportunities

Entirely optional

Not every potential treatment will be developed by WinQure Medicine.

Researchers, academic institutions and other therapy developers may be working on treatments that could be relevant to the patient's disease.

If you wish, RAREkey can also help make you aware of these opportunities.

Would you like the patient to be considered for therapeutic opportunities from collaborating researchers and therapy developers outside WinQure?(Required)

Yes

Choosing No will not affect registration with RAREkey or consideration for WinQure programs.

7. How your information is protected

We recognise that genetic information is particularly sensitive.

Genetic reports and other personal information submitted through RAREkey are securely stored and reviewed by WinQure Medicine. They are not publicly visible.

Information about patient communities may be used to help identify where treatments are needed and whether developing new therapies may be possible.

Where information is shared with external collaborators, WinQure takes appropriate measures to protect patient identities, particularly where very small patient numbers could make individuals identifiable.

Identifiable information will only be shared with external collaborators in accordance with applicable data protection legislation, the relevant permissions and WinQure's privacy documentation.

Genetic reports will not be shared with external researchers or therapy developers through RAREkey's optional external therapeutic opportunities process.

You can change your sharing preferences or withdraw consent at any time by emailing [email protected].

Withdrawal of consent does not affect the lawfulness of processing carried out before withdrawal. Following withdrawal, information will be deleted or otherwise handled in accordance with applicable legal obligations and the relevant privacy documentation.

For further information about how personal and genetic information is processed, including retention periods, data sharing, international transfers and data protection rights, please read:

WinQure Medicine Privacy Policy

WinQure Medicine Patient Information Notice

8. What happens next?

1. We receive your registration
Your information is securely submitted to RAREkey.

2. Our scientific team reviews your genetic information
We assess the genetic cause of the disease and whether an experimental therapy could potentially be developed.

3. We contact you if further information is needed
We may reach out to clarify your genetic test results or request additional details.

4. We contact you if a relevant opportunity arises
If we identify a potentially relevant therapeutic opportunity, we may contact you to discuss it.

This is also when we want to hear from patients and families about how the disease affects everyday life, what they would most like a treatment to improve and what would make participation in a clinical trial possible.

These experiences can help shape treatment priorities, the outcomes measured in clinical trials and how trials are organised.

You decide whether you wish to take part in any further discussions or programmes.

Registration does not mean that a treatment is currently available or that one will necessarily be developed.

Your voice matters, whether or not a treatment is currently available.
Every registration helps us better understand the needs of people living with ultra-rare genetic diseases.

RAREkey is a free patient registry operated by WinQure Medicine ApS.