Cure MFM13 Launches Patient Registry for Myofibrillar Myopathy Type 13 - Registration Now Open

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We are thrilled to announce that our Patient Registry - a Data Collection Program for individuals and families affected by Myofibrillar Myopathy Type 13 with Rimmed Vacuoles (MFM13) - is officially live and open for registration.

In partnership with RARE-X, a program of Global Genes, we have built a structured, secure database to collect standardized health information from patients and caregivers worldwide.

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About RARE-X and Global Genes

Global Genes is an international nonprofit organization supporting people living with rare diseases, founded in 2009. RARE-X, launched in 2021, is their nonprofit data-sharing and patient registry platform built specifically for rare disease communities. More than 120 rare disease organizations currently use the RARE-X platform.

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Why a Patient Registry Matters

MFM13 is an ultra-rare disease. Patient populations are small, data is scarce, and traditional clinical studies are difficult to conduct. Every single patient who contributes their information provides knowledge that cannot be gathered in any other way.

The registry collects Real-World Data (RWD) including symptoms, genetic findings, diagnosis history, mobility and daily functioning, quality of life, treatments, and other medical experiences reported directly by patients and caregivers. Data is collected over time, giving researchers a true picture of how MFM13 progresses throughout a patient's life.

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Your Data, Your Control

Your data belongs to you - always. As a participant, you decide how your data is used and you can withdraw at any time. While the registry is designed to be shared with qualified researchers to accelerate research, all access requests go through a review and approval process - researchers must apply and be accepted before they can access any data. The RARE-X platform provides full data governance, informed consent management, and security infrastructure.

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A Team You Can Trust

At Cure MFM13, we completed the Trusted Standard in Research, Ethics, Compliance & Safety Training (CITI Program), so you can be confident your data is handled responsibly and ethically.

What the MFM13 Data Collection Program aims to achieve:

• Inform researchers how MFM13 changes over time
• Build better data for use in clinical trials
• Give patients the opportunity to participate in future clinical trials
• Reduce the time needed to study new medicines
• Speed up access to treatments for patients
• Enable use of collected data as a control group (instead of actual patients) in clinical trials

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Watch: Webinar Recording - Introduction to the MFM13 Data Collection Program

On June 25, 2026, we hosted a webinar introducing the registry, walking through the RARE-X platform, and answering questions from the community.

If you missed the webinar or would like to rewatch it, the link to our YouTube channel is below.

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We Will Keep You Updated

Starting this registry is just the beginning. As data comes in, we will be sharing anonymous, aggregated insights from the registry on our social media channels - so the whole community can see what we are learning together. Follow us to stay informed as the picture of MFM13 starts to become clearer.

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How to Join

Registration takes place on the RARE-X platform. Visit: rare-x.org/mfm13

If you need help with the registration process, Ania and Sylwia are available to assist directly:

• Ania Kordala: ania@curemfm13.org
• Sylwia Szwec: sylwia@curemfm13.org

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Learn more

To learn more check Join the Registry.

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