Exciting News!

A groundbreaking gene therapy for Duchenne muscular dystrophy has been expanded by the FDA for children ages 4 and up. CHKD’s Chief of Neurology Dr. Crystal Proud, one of the world’s leading researchers on this treatment, is excited to offer it to more patients. Read more.

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Duchenne Muscular Dystrophy Program

CHKD's Duchenne muscular dystrophy program provides interdisciplinary care to patients in our region and beyond. We are nationally recognized as a Muscular Dystrophy Association Care Center and are certified by Parent Project Muscular Dystrophy, which recognizes excellence in the care of patients with muscular dystrophy. Through our comprehensive program, your child will be cared for by experts in neurology, physical medicine and rehabilitation, and pulmonary medicine.  

Duchenne muscular dystrophy is a genetic neuromuscular disorder that causes the body to weaken as the muscles break down and are replaced with fatty deposits. The disorder, which mostly affect boys, can lead to heart problems, scoliosis, and obesity.

Duchenne muscular dystrophy, and other types of muscular dystrophy, require life-long management. Your child’s care team will work with you to improve your child’s quality of life and provide the support you need to best care for your child’s needs.


Signs of Muscular Dystrophy and Treatment Options

Early Signs of Muscular Dystrophy

Muscular dystrophy is diagnosed between 3 and 6 years of age. Early signs include:

  • Delayed walking
  • Difficulty getting up from a sitting or lying position
  • Frequent falling and clumsy movement
  • Leg pain
  • Enlarged calves
  • Weakness in arms and shoulders

Treatments

There is no known cure for Duchenne muscular dystrophy. The goal of treatment is to control symptoms and improve quality of life.

Treatment may include:

  • Physical therapy
  • Positioning aids
  • Braces and splints
  • Medications

Steroid medications may also help slow the progression of the disease. These treatments may include:

  • Albuterol - a drug used for people with asthma
  • Amino acids
  • Carnitine
  • Coenzyme Q10
  • Creatine
  • Fish oil
  • Green tea extracts
  • Vitamin E


Advanced Care - Novel Therapeutics and Gene Therapy

Specializing in neuromuscular conditions, Neuromuscular Neurologist Dr. Crystal Proud is fiercely committed to ensuring CHKD provides the latest treatments. Thanks to her commitment, CHKD offers a new FDA-approved treatment, Elevidys, that presents a real hope to patients with Duchenne muscular dystrophy.

Elevidys is a gene therapy that spurs the development of a protein similar to the protein missing in the muscle cells of children with Duchenne. This therapy is administered in a single intravenous dose. Email Gene.Therapy@CHKD.org for more information or to request a consultation.