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Treatment of muscular dystrophy

Muscular dystrophy is a group of diseases that cause progressive weakness and loss of muscle mass. In muscular dystrophy, abnormal genes (mutations) interfere with the production of proteins needed to form healthy muscle.

Right now, there’s no cure for the disease. But there are many treatments that can improve symptoms and make life easier for you and your child.

Your doctor will recommend a treatment based on the type of muscular dystrophy your child has. Some of them are:

  • Physical therapy uses different exercises and stretches to keep muscles strong and flexible.
  • Occupational therapy teaches your child how to make the most of what their muscles can do. Therapists can also show them how to use wheelchairs, braces, and other devices that can help them with daily life.
  • Speech therapy will teach them easier ways to talk if their throat or face muscles are weak.
    Respiratory therapy can help if your child is having trouble breathing. They’ll learn ways to make it easier to breathe, or get machines to help.
  • Medicines can help ease symptoms. They include:
    • Eteplirsen (Exondys 51), golodirsen (Vyondys53), and vitolarsen (Viltepso) for treating DMD. They are injection medications that help treat individuals with a specific mutation of the gene that leads to DMD, specifically by increasing dystrophin production. Talk to your child’s doctor about possible side effects.
    • Anti-seizure drugs that reduce muscle spasms.
    • Blood pressure medicines that help with heart problems.
    • Drugs that turn down the body’s immune system, called immunosuppressants; they may slow damage to muscle cells.
    • Steroids like prednisone and defkazacort (Emflaza) that slow down muscle damage and can help your child breathe better. They can cause serious side effects, such as weak bones and a higher risk of infections.
    • Creatine, a chemical normally found in the body, that can help supply energy to muscles and improve strength for some people. Ask your child’s doctor if these supplements are a good idea for them.
  • Surgery can help with different complications of muscular dystrophy, like heart problems or trouble swallowing.

Scientists also are looking for new ways to treat muscular dystrophy in clinical trials. These trials test new drugs to see if they are safe and if they work. They often are a way for people to try new medicine that isn’t available to everyone. Your doctor can tell you if one of these trials might be a good fit for your child.

Taking Care of Your Child

It’s hard when your child loses strength and can’t do the things other kids can do. Muscular dystrophy is a challenge, but it doesn’t have to keep your child from enjoying life.

There are many things you can do to help them feel stronger and get the most out of life.

  • Eat right. A healthy, well-balanced diet is good for your child in general. It’s also important for helping them stay at a healthy weight, which can ease breathing problems and other symptoms. If it’s hard for them to chew or swallow, talk to a dietitian about foods that may be easier to eat.
  • Stay active. Exercise can improve your child’s muscle strength and make them feel better. Try low-impact activities like swimming.
  • Get enough sleep. Ask your doctor or therapist about certain beds or pads that can make your child more comfortable and rested.
  • Use the right tools. Wheelchairs, crutches, or electric scooters can help your child if they have trouble walking.

The disease will most likely have a big impact on your family. Remember that it’s OK to ask a doctor, counselor, family, or friends for help with any stress, sadness, or anger you may feel. Support groups are also good places to talk to other people who have lived with muscular dystrophy. They can help your child connect with others like them and give you and your family advice and understanding.


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