Myotonic dystrophy type 3

Common Name(s)

Myotonic dystrophy type 3, DM4, DM3, Myotonic dystrophy type 4

Myotonic dystrophy type 3 and type 4 (DM3 & DM4) are possibly two other forms of myotonic dystrophy. There are a small number of people with symptoms fitting a diagnosis of myotonic dystrophy but who do not have mutations in the genes which cause DM1 and DM2. At this time, medical researchers and specialists are uncertain whether these are truly new forms of myotonic dystrophy. It may be that mutations or changes in other genes may cause these unique forms. It may also be that these individuals actually have a different condition but have a unique presentation (different symptoms than normally seen in that condition).

In general, symptoms of myotonic dystrophies may include progressive muscle loss and weakness, particularly in the lower legs, hands, neck, and face. People with myotonic dystrophy often have prolonged muscle tensing (myotonia) and are not able to relax certain muscles after use. The symptoms may vary greatly even within the same family.

Although a diagnosis is very important, even with a diagnosis of DM1 or DM2, it may be difficult to make predictions of how the condition will affect your life over time since the condition can affect each person differently. It is therefore very important to learn as much as you can about myotonic dystrophy, the different types and the different symptoms and complications you may experience. You will most likely need to see a number of different specialist - understanding who is the best doctor to see for your different symptoms is very important. If you have been diagnosed with DM3 or DM4, talk with your doctor about which specialists you should see. Connecting with a support organization is often a great way to learn more about your diagnosis and how to get the treatment and care you may need.

Source: Advocacy organizations associated with the condition.

 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Myotonic dystrophy type 3" for support, advocacy or research.

Myotonic Dystrophy Foundation

The Myotonic Dystrophy Foundation (MDF) exists to enhance the quality of life of people living with myotonic dystrophy (DM), and to maximize research efforts focused on finding treatments and a cure for this disease, through community support, education, advocacy and research. Community Support & Education - MDF programs and services provide emotional support and education to help affected individuals and their families, and information and resources to raise awareness about DM in the medical community and the general population. Advocacy - Through MDF-sponsored initiatives and in partnership with other rare disease organizations, the Foundation advocates for increased funding for DM research and patient support services. Research - MDF raises funds to accelerate understanding of DM through research. MDF supports both clinical and scientific research targeted to finding treatments and eventually a cure for this chronic, degenerative disease.

Last Updated: 20 Apr 2015

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General Support Organizations

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Myotonic dystrophy type 3" for support, advocacy or research.

Myotonic Dystrophy Foundation

The Myotonic Dystrophy Foundation (MDF) exists to enhance the quality of life of people living with myotonic dystrophy (DM), and to maximize research efforts focused on finding treatments and a cure for this disease, through community support, education, advocacy and research. Community Support & Education - MDF programs and services provide emotional support and education to help affected individuals and their families, and information and resources to raise awareness about DM in the medical community and the general population. Advocacy - Through MDF-sponsored initiatives and in partnership with other rare disease organizations, the Foundation advocates for increased funding for DM research and patient support services. Research - MDF raises funds to accelerate understanding of DM through research. MDF supports both clinical and scientific research targeted to finding treatments and eventually a cure for this chronic, degenerative disease.

http://www.myotonic.org

Last Updated: 20 Apr 2015

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General Support Organizations

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Myotonic dystrophy type 3" returned 2 free, full-text research articles on human participants. First 3 results:

Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1.
 

Author(s): Chad R Heatwole, Katy J Eichinger, Deborah I Friedman, James E Hilbert, Carlayne E Jackson, Eric L Logigian, William B Martens, Michael P McDermott, Shree K Pandya, Christine Quinn, Alexis M Smirnow, Charles A Thornton, Richard T Moxley

Journal: Arch. Neurol.. 2011 Jan;68(1):37-44.

 

To evaluate the safety and tolerability of recombinant human insulin-like growth factor 1 (rhIGF-1) complexed with IGF binding protein 3 (rhIGF-1/rhIGFBP-3) in patients with myotonic dystrophy type 1 (DM1).

Last Updated: 11 Jan 2011

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Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3.
 

Author(s): Alexei Pushechnikov, Melissa M Lee, Jessica L Childs-Disney, Krzysztof Sobczak, Jonathan M French, Charles A Thornton, Matthew D Disney

Journal: J. Am. Chem. Soc.. 2009 Jul;131(28):9767-79.

 

Herein, we describe the design of high affinity ligands that bind expanded rCUG and rCAG repeat RNAs expressed in myotonic dystrophy type 1 (DM1) and spinocerebellar ataxia type 3. These ligands also inhibit, with nanomolar IC(50) values, the formation of RNA-protein complexes that ...

Last Updated: 15 Jul 2009

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Myotonic dystrophy type 3" returned 0 free, full-text review articles on human participants.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

There are currently no open clinical trials for this condition.