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Myotonic Dystrophy Family Registry

Myotonic Dystrophy Family Registry

Recruiting
All
Phase N/A

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Overview

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.

Description

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) such as disease symptoms and demographic information to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.

The Registry supports trials and studies, making it easier for researchers to explore data and identify possible trial and study participants. It is the first DM registry that gives community members the opportunity to explore anonymous Registry data, to see what the DM community looks like and what others with DM experience. It also provides information on the community of people living with DM, giving researchers and other medical professionals the opportunity to improve how they treat those affected with DM and learn more about how and why certain treatments work and don't work.

Eligibility

Inclusion Criteria:

  • Diagnosed with congenital, juvenile-onset or adult onset DM1 or DM2 (confirmed by clinical exam or genetic test)

Exclusion Criteria:

  • Not diagnosed with DM, unaffected family members

Study details
    Myotonic Dystrophy
    Congenital Myotonic Dystrophy
    Myotonic Dystrophy 1
    Myotonic Dystrophy 2
    Dystrophia Myotonica
    Dystrophia Myotonica 1
    Dystrophia Myotonica 2
    Myotonia Dystrophica
    Myotonic Dystrophy
    Congenital
    Myotonic Myopathy
    Proximal
    PROMM (Proximal Myotonic Myopathy)
    Proximal Myotonic Myopathy
    Steinert Disease
    Steinert Myotonic Dystrophy
    Steinert's Disease
    Myotonia Atrophica

NCT02398786

Myotonic Dystrophy Foundation

26 January 2024

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FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

Do I get compensated for taking part in clinical trials?

Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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