Imagine a dedicated system designed to gather information about individuals living with a particular disease, like cancer or diabetes, or even an uncommon condition like MdDS. Imagine the collected data being used by researchers and clinicians to better understand the disorder and see what treatments are actually working – and possibly change the future of patient care. That’s what a patient registry is and can do for you!
Powered by the National Organization for Rare Disorders‘ IAMRARE® program, the MdDS Patient Registry will be the first longitudinal research study to collect MdDS-specific patient data. The more people who participate means more meaningful data. Discoveries made through this study can pave the way to advancements in additional research and better patient outcomes.
Study Aims
The goal of the MdDS Patient Registry will be to help researchers, clinicians or industry partners learn more about MdDS. The information collected in the MdDS Patient Registry will be used to study MdDS with the following major goals:
- Describe People with MdDS: Understand who develops MdDS and how symptoms can be different for each person. By tracking many individuals, we can better understand the typical course of MdDS, its various characteristics, and how it’s generally managed by clinicians.
- Understand MdDS Over Time: Learn how MdDS develops, what it feels like, and how symptoms change longitudinally (over time). By following the same people over time, this registry can reveal how things change, or stay the same, for individuals.
- Learn About Diagnosis and Treatment: Find out how MdDS is diagnosed, what treatments are available, and how people respond to them. A registry can show us how treatments perform in the real world, with all the complexities that come with it.
- Improve Care for People with MdDS: Use what we learn to provide better support and treatment for those with MdDS. The rich data within a registry is a goldmine for researchers who are working to understand MdDS, develop new therapies, and spot important trends.
- Identify Participants for Research: Find people with MdDS who may want to join future research studies or clinical trials.
Your Data, Your Impact
This study will offer you the opportunity to make a meaningful contribution toward improving the lives of those affected by MdDS. When you join, you’ll be asked to share information about your symptoms and overall MdDS experience. You may be wondering how sharing your information helps MdDS research. Or what happens to your data? Click or tap to reveal answers to your questions.
How will my data be collected?
The registry uses easy-to-complete surveys and questionnaires which are online instruments. Housed on the IAMRARE® platform, industry-standard safeguards to protect your information and privacy are in place.
Who can see my data?
Rest assured that maximum security and privacy practices will be followed. Data may be provided to approved researchers. The goal is to provide these researchers with the minimum data necessary to accomplish their research study. Data shared with outside researchers will be de-identified [Protected health information (PHI) will be hidden] where possible. This means your identity is private and protected.
How will my data be used?
The information you share in the registry will be used by:
• Researchers: To study MdDS and identify potential treatments.
• Clinicians: To improve care for people with MdDS.
• The MdDS Foundation: To advocate for awareness, research funding, and patient resources.
These groups work together with a single goal: to improve the lives of people living with MdDS.
Who can participate?
If you’ve experienced symptoms of MdDS, the perception of motion, for at least 30 days, you will be able to enroll when the registry launches. Study enrollees may designate a Legally Authorized Representative (LAR) to complete surveys and questionnaires on their behalf.
How easy is participation?
Very. The main survey, MdDS Status, will collect answers about your symptoms, balance, dizziness, and quality of life. Each area of focus (or domain) is completed separately, and at your convenience. Supplementary questionnaires cover aspects like medical and family histories. They’re extremely easy to complete, and you can stop anytime you like. The system will save your responses for you.
When can I start?
We anticipate the registry launching the summer/fall of 2025. To be among the first to be alerted, sign up for our newsletter using the form at the bottom of the page.
Provide your email address, and we’ll notify you when the registry is ready for participation!
Coming Soon — a visual guide to the study! You’ll see how the study is designed to flow and also discover benefits of participation.
Glossary of Terms
The registry may use terminology you’re not familiar with. Click or tap to expand/collapse the definitions.
Advisory Board
A committee comprised of clinicians and data scientists, patient advocates, and patient ambassadors. The Registry Advisory Board is responsible for reviewing all requests for registry data to ensure that the proposed project has scientific merit and will be valuable to the community. They will also be responsible for deciding what specific data will be given to researchers. This will be limited to only the data needed to answer the researcher’s question. Whenever possible, the data will be de-identified.
De-identified Data
De-identification means personal data such as name, dates and address will be removed. Instead a randomly assigned identification code will be used.
Domain
Living with a chronic disorder can affect every aspect of your life, from how you move to how you feel emotionally. With the MdDS Patient Registry, researchers will study these effects in separate areas, or domains, including balance, dizziness, and quality of life. By measuring domains separately, we can get a clearer picture of how MdDS – a disorder of perceived motion — impacts individuals and find better ways to manage it.
Informed Consent
A process in which patients are given important information, including possible risks and benefits, about a medical procedure or treatment, genetic testing, or research. This is to help them decide if they want to be treated, tested, or take part in the research. Our registry’s consent document follows the framework provided by federal regulations.
Institutional Review Board (IRB)
An Institutional Review Board (IRB) is an independent group of people that reviews research proposals to make sure they properly protect participants. An IRB has reviewed the MdDS Patient Registry to ensure that it meets ethical and regulatory standards.
Longitudinal Study
A type of study where researchers repeatedly collect data from the same group of individuals over an extended period of time. It follows the same participants throughout the study to analyze how things evolve over time. This allows researchers to observe and track changes and developments in variables like behaviors, health outcomes, or opinions across different points in time.
Modifier
Personal activities and environmental factors can influence MdDS symptoms, acting as modifiers that either increase or decrease the sensation of motion. The effect of these modifiers is unique to each individual. To discover common modifiers that exacerbate or alleviate symptoms, please visit the
Symptoms page.
Natural History
The course a disease takes, over time (longitudinally), is its natural history. Collection of individual patient data, repeatedly over time, illustrates the progression of that individual patient’s condition. Collecting data about the progression of MdDS, across the MdDS community, is one of the most impactful and consequential contributions we can make in working towards the development of treatments and cures.
Principal Investigator
A principal investigator (PI) is the primary person responsible for the design and conduct of a research project or clinical trial. A co-investigator(s) may share responsibility.
Protected Health Information (PHI)
Protected health information (PHI) is information that may be used to identify an individual.
Research Study vs. Clinical Research or Study
A research study aims to gain knowledge about a phenomenon, while clinical research aims to develop new treatments, diagnostics, or preventative measures by testing them in humans. Our Patient Registry is a research study designed to collect MdDS-specific patient data. For more detailed information on the study design,
read this article.
Statistical Significance
Researchers and clinicians need reliable data. When data is collected from too small a sample, it is unstable because the margin for error is too great. For data to be statistically significant, certain thresholds must be met. Using our support group as a survey population, if 100 of the 5,000 active members completes a survey, the margin of error will be ~10%. If 1,000 of the 5,000 active members completes a survey, the margin of error will be less than 3%. Your participation in the patient registry can make a meaningful difference!
Remission
Patients with 0 perception of motion, sustained for at least six (6) months, are considered in remission.
Translational Research
This type of research aims to bridge the gap between basic laboratory findings and clinical applications, essentially “translating” discoveries made in the lab into treatments or interventions that can directly benefit patients in a clinical setting. It can take research from the bench to the bedside to improve human health.
Have questions about the IAMRARE® platform? This page provides an introduction the MdDS Patient Registry and gives definitions for terminology used with it. A comprehensive FAQ for the IAMRARE platform can be found on NORD®’s IAMRARE site.
