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All fields are optional. Signing up for the registry will also place you on the Cure CMD newsletter list.
What is a registry?
A patient registry is a database that contains your contact information, medical record and answers to an annual health survey.
What is the purpose of a registry?
A registry allows you to connect with current research trials. Without a patient registry, a clinical trial cannot proceed. A clinical trial to test a therapy or cure will require participants (people like you). See Research page
How will I get contacted?
Protecting your information is of critical importance to Cure CMD. You will be contacted by Cure CMD by email or letter if you meet the criteria to participate in a clinical trial. Being contacted, does not obligate you to participate. You may then contact the principal investigator of the clinical trial to get more information and establish a relationship.
If I decline to participate in a clinical trial, does this mean that I won’t be notified in the future?
Declining to participate is your right and will not preclude you from being notified in the future if once again you meet criteria to enter a clinical trial. At the same time, one of the biggest impediments to accomplishing our goal of finding a cure or therapy, is the limited number of trial participants given how rare CMD is. Each time a participant chooses not to participate in a clinical trial, it decreases the possibility that the clinical trial will go forward.
Who will have access to my information?
Cure CMD will select an advisory committee to oversee access to the registry. The committee’s function will be to provide support to the genetic counselor who interacts with you to verify your medical information, help direct you towards a genetic diagnosis and answer your questions.
Your information will be filtered to remove any personal identifiers and make the information HIPAA compliant. The committee will review applications to access a de-identified version of the patient registry based on stated purpose and use. A typical person who might apply to access the registry would be a biostatistician, neurologist or genetic counselor who seeks to better characterize the disease based on answers to health surveys.
What are the benefits to me of joining the CMD registry?
The benefits of joining the CMD registry are:
- Connecting with clinical drug trials that hold the promise of a new therapy or cure for CMD
- Maintaining a record of your health and medical records
- Contributing to better CMD disease awareness
- Helping turn clinical trials from a possibility into a reality
- Receiving help from a licensed genetic counselor to answer questions regarding the annual health survey, how to enter your medical record and where to go for genetic testing
What is my responsibility?
Joining the CMD patient registry, means you are responsible for your information and to the promise of finding a therapy or cure. You are responsible for contacting Cure CMD if your contact information changes or there are additional medical records that have become available. You are responsible for completing the annual health survey, verifying your current health status. You may share your health survey with your physician if you have any questions. You may also contact the genetic counselor if you have any questions.
Will the registry be international?
The CMD International Patient registry (CMDIR) will be an international patient self report registry. This means that people with CMD and parents of young children with CMD will answer a core set of questions. These questions will be translated into 4 core languages: English, French, Spanish and Japanese. The CMDIR will act as a central coordinating effort to count the number of people in the world with CMD, assist people with confirmation of genetic disease where possible and connect people with CMD to future clinical trials.
What is a biorepository?
A tissue respository is a lab that houses blood and tissue specimens (fibroblasts and muscle biopsies). Its goal it to preserve specimens for future research needs. Specimen identifiers are safeguarded. Applications to use specimens are reviewed by an expert committee as designated by an IRB approved process. A biorepository helps researchers test hypotheses and test drug compounds in living human tissue.
How do I participate in a biorespository?
The best way to participate in a biorespository is to forward a part of a skin, muscle or blood sample to the Cure CMD biorespository, once it is established. This does not mean that you need to get your blood drawn tomorrow, or schedule a muscle biopsy. However, if you are having a procedure done, you might consider contacting Cure CMD to find out how to donate part of the specimen that is not being used for testing and could be frozen.
How does a biorespository benefit me?
The benefit of sending part of your blood, skin or muscle tissue to a biorepository is to provide researchers with living material to study a drug in your tissue. The researcher will not know it is your tissue, because the material will be de-identified. However the researcher will receive information about your type of CMD, age and any medical therapies you have tried. This benefit means that if a drug has an effect, you have contributed to finding a drug that might work for your type of CMD and possibly for other types of CMD as well.
What is a natural disease progression study and how does this type of study validate outcome markers?
A natural disease progression study is a data entry system that records specific markers of disease and disease progression to track patients with all forms of CMD. This type of study will benefit the CMD community, by improving general medical knowledge about these rare diseases, including the number of affected individuals. It will also help establish guidelines for interventions at critical junctures during disease progression.
The goal is to set up natural disease progression studies and evaluate outcome markers to validate them for future clinical trials. The outcome markers are tests that can be performed in the clinic setting, such as testing muscle strength or breathing strength. These markers first need to be validated in CMD and then can be used when testing a new drug to demonstrate if it does or does not work in CMD.
Biomarkers are another type of outcome marker. Biomarkers are usually proteins in the blood that can go up or down in a disease depending on disease progression. Using a biomarker is another way to test if a drug does or doesn’t work. There are currently no biomarkers for CMD.
