Muscular Dystrophies and Myopathies

Muscular dystrophy and myopathy symptoms

The main symptom of a muscular dystrophy or myopathy is muscle weakness.

Depending on the type, weakness may stay the same or slowly get worse. Symptoms can be different depending on when they start.

Common symptoms include:

• Trouble walking
• Difficulty using the arms for daily tasks, like brushing hair or lifting things
• Stiff or tight muscles and joints
• Heart problems
• Breathing or coughing problems
• Trouble swallowing
• Weak or brittle bones
• Differences in brain development or learning

Each child’s symptoms and how quickly they change can be different.

Types of muscular dystrophies and myopathies studied at St. Jude Children’s Research Hospital include:

• Duchenne muscular dystrophy (DMD)
• Limb-girdle muscular dystrophy (LGMD)
• Facioscapulohumeral dystrophy (FSHD)
• Nemaline myopathy

About Duchenne muscular dystrophy (DMD)

DMD is the most common type of muscular dystrophy. It affects mostly boys. Symptoms usually start between ages 2 and 5. 

Children with DMD often have trouble walking or climbing stairs. Over time, they may need a wheelchair. DMD also affects the heart and breathing muscles. It is important to see the health care team often.

DMD occurs in about 1 in 3,500 to 5,000 babies from all ethnic groups. 

Read more about DMD.

About limb-girdle muscular dystrophy (LGMD)

Limb-girdle muscular dystrophies (LGMDs) are rare muscle diseases that affect both boys and girls. These conditions usually affect muscles around the hips and shoulders first. These are called the “limb girdle” areas. 

Some types of LGMD start in childhood. Others start later in life.

People with LGMD may have mild symptoms. Others may have more serious problems with walking, lifting, or using their arms. Some people may develop disabilities. 

LGMD is less common than DMD. It affects fewer than 2 out of every 100,000 people. The numbers vary by type and area of the world.

How muscular dystrophies are treated

Right now, there is no cure for muscular dystrophy. Some genetic treatments are available for DMD, and more are being tested for some of these disorders. 

Your child’s care team will work to manage symptoms and help your child maintain function.

Muscular dystrophies and myopathies care at St. Jude

St. Jude is working to better understand rare, catastrophic genetic neurological disorders in children. Our goal is to change how these disorders are treated. The Center for Experimental Neurotherapeutics (CENT) was started at St. Jude as part of the Pediatric Translational Neuroscience Initiative (PTNI)  to focus on these neurological disorders.

Children with muscular dystrophies and myopathies who are accepted at St. Jude may take part in clinical research or clinical trials, as they become available. These studies help scientists learn more and develop new treatments. 

Muscular dystrophies and myopathies clinical research

Resources outside St. Jude