Accelerate drug development in MMA

Important research records are trapped in hospitals across the country. As a result, drug and gene therapy researchers can’t access the information they need to advance treatments.

But as a patient, you can. Unlock your records and contribute them to research, privately and securely.

RDMD

Questions? Check out our FAQ

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By joining the MMA program, you will help advance new research. If you know other patients or families who may be interested, you can help by sharing this link.
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Why join the MMA program

For the future

For the future

Help researchers get the information they need to advance development of new drug and gene therapies.

For the MMA community

For the MMA community

Jumpstart research and increase knowledge and understanding of MMA.

For you

For you

See all your records in one place. Track how you are contributing to research and be notified about new clinical trials.

How it works

Sign up

1. Sign up

Create your private account and review our research consent (takes about 10 minutes)

We get to work

2. We get to work

We do all the behind-the-scenes work to retrieve and process your records

Empower research

3. Empower research

Track your contribution to research projects and, based on your records, find out if you qualify for new clinical trials based on your records

Be part of research

Questions? Check out our FAQ

Our values &
principles.

1
Patients own their data

We believe that the only way to keep data accessible to the researchers who need it is by enabling patients to own and control their own data. Patients decide for themselves whether they want to privately and securely contribute to research, rather than having the decision made on their behalf.

2
We protect patient privacy

We abide by a strict research consent and privacy policy. We only share de-identified data with researchers, foundations, and therapeutic companies with patients’ consent. Protecting the privacy of patients and ensuring the security of their information is our highest priority.

3
Researchers gain access

We aim to make useful data accessible to all researchers who have legitimate research questions or research programs. For academic research, we offer access without charge. We take research ethics seriously, and we have policies in place to ensure that research is conducted with integrity.

4
Inclusiveness in the community

We don’t like to take sides—we give all members of the community the option to get involved. Success in rare disease research takes a village and we always welcome additional collaborators who can advance and benefit from the mission.

5
Driving forward therapeutic development

Our primary mission is to accelerate drug development, so we prioritize generating insights specifically relevant to therapeutic research. We invest deeply in quality, security, and compliance to make this a reality.

Who’s involved?

Gregory Enns - Stanford
Gregory Enns - Stanford

Our work with MMA research

Methylmalonic acidemia (MMA) is a genetic condition where individuals cannot break down certain proteins and fats. The symptoms of MMA typically begin very early in life and vary from mild to severe. Those with MMA can experience vomiting, dehydration, fatigue, low muscle tone, and difficulty gaining weight. Developmental delay, intellectual disability, and damage to the kidneys, pancreas, liver, and brain can also occur. MMA requires lifelong management and in some cases, can be life-threatening. There is currently no cure.

RDMD drives research on your condition as well as research on general health and wellness. These are the current research projects we aim to complete in order to increase our understanding of methylmalonic acidemia and drive drug development and clinical trials.

  • Jumpstart
  • Accelerate
  • Insights

The MMA program is in the jumpstart stage.

Be part of research

Natural History Study

With early diagnosis, the expected lifespan of patients with MMA has increased over time. By studying the medical chart, researchers will be able to understand the issues that MMA can cause over a person’s lifespan. This will help researchers learn about the major issues that new MMA treatments should address in the future.

    

MMA Subtypes Study

There are several different categories of MMA. Some of these categories, such as the “mut0” or “mut-,” are more common than others. It is important to understand how each MMA category is similar to and different from the others over a person’s lifespan. By studying this, researchers will learn how we can better prepare families for the future and learn how different categories of MMA could be treated.

  

MMA Treatment Outcomes

While there is currently no cure for MMA, patients are often managed by diet, carnitine supplementation, antibiotics, and other treatments. Additionally, some patients receive an organ transplant. Learning more about why certain approaches are tried and how well they worked for each patient will help researchers understand what types of therapies might work for MMA in the future.

   

Hackathon & Discovery

Analyzing the genetic data of patients with MMA could help researchers understand why some patients have very mild or very severe MMA, and also help them to pinpoint different ways to treat the disease.