Adult-Onset MMD1/MMD2 and Juvenile-Onset MMD1

Medical Management

This section addresses medical management of the many symptoms of adult-onset MMD1 and MMD2, as well as juvenile-onset MMD1. These three forms of MMD share similar medical management strategies.

The subjects covered are:

Muscle Physiology — Masahiro Iwamoto, Ph.D., D.D.S.

Masahiro Iwamoto, research scientist at the Children’s Hospital of Philadelphia and associate professor of pediatric orthopedics at the University of Pennsylvania School of Medicine, was awarded an MDA research grant totaling $405,000 over a period of three years to study new ways to reduce muscle degeneration.

Why Does It Take So Long To Go from Mouse to Man?

John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human clinical trials. Why does it take so long?

International MMD Consortium Includes Professionals, Families

The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and MMD1, also known as DM1 and DM2).

Medical Management

As yet, there’s no specific treatment that “gets at the root” of type 1 or type 2 myotonic muscular dystrophy (MMD1 or MMD2). Treatment is aimed at managing symptoms and minimizing disability.

This section first addresses medical management of the many symptoms of adult-onset MMD1/MMD2 and juvenile-onset MMD1. Not everyone will require all these aspects of medical management, and some symptoms may first appear or worsen as a person grows older.

Description: 

MDA leads the search for treatments and therapies for myotonic muscular dystrophy (MMD). The Association also provides comprehensive supports and expert clinical care for those living with MMD.

In this section, you’ll find up-to-date information about myotonic muscular dystrophy, as well as many helpful resources. This information has been compiled with input from researchers, physicians and people affected by the disease.

Research Briefs: FA, MG, MM, MMD1, gene therapy

Edison drugs target FA, mitochondrial diseases

Going to the Emergency Room: Tips for People with Neuromuscular Diseases

When a medical emergency strikes — and the patient is a person with a neuromuscular disease— it’s not just getting to the emergency room quickly that’s critical. It’s also critical to ensure the ER staff understands the patient’s special needs caused by muscle disease.

Care Guidelines Issued for Congenital Muscular Dystrophies

Update (January 25, 2012): This story was updated to reflect the online availability of a CMD Family Guide.

The Brain in MMD

*Many people interviewed for this article asked not to be identified to protect the privacy of affected family members.

“Everybody knows the word apathy,” says a California woman whose 25-year-old daughter’s type 1 myotonic dystrophy (MMD1, sometimes called DM1) was diagnosed just a few years ago. “People use the word loosely. I don’t think it does justice to the reality of this disease.”

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