Muscular dystrophy (MD) is a group of genetic conditions that cause increasing muscle loss. Children's Health℠ has one of the most comprehensive pediatric MD teams in Texas, caring for kids from diagnosis through their transition to adult care.
While there’s no cure for muscular dystrophy in children, we provide the latest therapies to manage symptoms and help slow the course of the disease. Together, we help children retain their strength, prepare for changes and live their healthiest life.
When a child has muscular dystrophy, their body doesn’t produce the proteins they need to form healthy muscles. Muscle cells die and are replaced by fat cells. At Children’s Health, our combined expertise of specialists from neurology, cardiology, pulmonology, rehabilitation and other disciplines coordinate patient care.
Children’s Health is home to the only Duchenne muscular dystrophy (DMD) clinic in Texas, certified by the Parent Project Muscular Dystrophy as offering best practices in care.
We prioritize normalizing everyday tasks and experiences like getting ready for school, eating, playing with friends and getting around the community.
Different types of muscular dystrophy in children progress and affect muscles in different ways:
DMD is the most common form of muscular dystrophy in children. It usually starts between ages 2 and 5, and it typically affects boys. Children’s Health has the only certified Duchenne Muscular Dystrophy Clinic in Texas.
This form usually starts when a child is a teenager, and it develops more slowly than DMD.
This type of muscular dystrophy can start in childhood or early adulthood. Myotonia means that muscles have a hard time relaxing after they have contracted.
This form progresses slowly. Usually, it first affects muscles in the shoulders, back and hips.
This type usually appears in the later teen years and tends to progress slowly. It usually affects the muscles of the face, back and upper arms.
This muscular dystrophy appears in early adulthood. It usually affects the muscles of the face and neck first.
Symptoms vary depending on the type of muscular dystrophy, but a child may experience:
At Children’s Health, your doctor will do a physical exam and ask about any problems your child is having. They may also ask about your family health history and will do tests to rule out other conditions. If the doctor suspects muscular dystrophy, they will take a blood sample and test for high levels of an enzyme called creatine kinase, which is a sign of muscular dystrophy.
Next, doctors will take a blood sample and do a genetic test (called a genetic panel) to try to identify the specific type of muscular dystrophy. Your doctor also may do a muscle biopsy, which means taking a small sample of your child’s muscle tissue and looking at it under a microscope.
Certain genes help make the proteins your body needs to build healthy muscles. Muscular dystrophy happens when a change (mutation) in those genes causes them to not work properly. Each type of muscular dystrophy is caused by a mutation to a specific gene. These genetic changes usually are inherited (passed down from parent to child), but sometimes they happen on their own.
Although there currently is no cure for muscular dystrophy in children, our care team will work with your child to improve and strengthen how their muscles and joints work, and to slow the muscle loss. This care can help extend the time that they can stay active and independent.
Muscular dystrophy usually gets worse over time, and it can affect different organs. As your child passes through different stages, they may need different kinds of treatment. Our care team will closely monitor your child to ensure that they get the care they need at every stage.
We may prescribe steroids for your child, to help reduce the inflammation that can come with muscle loss and to slow down the progression of the condition. There are also several approved medications that are given for specific types of muscular dystrophy.
Your child may receive care from different specialists, depending on their needs. This includes:
Researchers at Children’s Health and other sites are working together to develop new ways to treat muscular dystrophy, including pursuing genetic therapies that would treat its cause. Some research focuses on creating artificial genes to replace the defective ones so that cells throughout the body could produce the missing proteins needed for healthy muscles.
At Children’s Health, your child can see specialists who can help with their treatment.
The life expectancy is different based on the type of muscular dystrophy your child has. For children with duchenne muscular dystrophy (DMD), better care for the heart and lungs has meant that children are living beyond their teen years and into their 30s and even their 40s.
Different types of muscular dystrophy appear at different ages. Symptoms may start early in childhood or as late as adulthood.
Muscular dystrophy is a progressive condition that needs life-long management. Walking, sleeping and eating often become more difficult over time.
For some types, including Duchenne muscular dystrophy, children will eventually need a wheelchair because their leg muscles are too weak to walk and support them. Heart or lung problems are also common for many patients. Some patients eventually need eating and breathing support.
At Children’s Health, we prioritize normalizing everyday tasks and experiences like getting ready for school, eating, playing with friends and getting around the community. Our interdisciplinary team will work with your child to help them comfortably maintain as much physical independence as possible.
Life at home might not change too much in the beginning. As your child's needs change, they will likely need some tools to help them move around the house safely, eat, bathe and sleep. Your child’s doctors will help you prepare for new support systems like wheelchairs, braces, modified utensils and for some communication tools. They will also help you and your child learn to use them.
Your Children’s Health team can also do home assessments and will work with your school to make sure your child’s needs are being met there as well.