TREATMENT IS A
EMFLAZA® is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older.
A strong team of medical specialists and caregivers is key to effective treatment of Duchenne muscular dystrophy. It’s important to have a range of specialists from various disciplines for support and to carefully manage the condition as well as monitor any potential side effects.1
Be sure to help your child stick to a healthy lifestyle: a well-balanced diet, watching sugar and salt, and adding vitamin supplements when needed.1
It’s important to organize your team early and keep them involved, particularly your neurologist, to meet the needs of your child.
MEET YOUR HEALTHCARE TEAM1-3
The earlier your child receives an accurate Duchenne diagnosis, the better off he will be. It’s also important to have an ongoing assessment of your child’s condition because, as Duchenne progresses, his treatment needs might change.
A care coordinator or social worker can work with you to transition the care of your child across his lifespan. To make sure his needs never go unmet, transition planning should include continuous care with pediatric doctors until adult care is needed.
PRIMARY CARE &
While your child’s primary care physician is typically fully aware of your child’s medical history, emergency care personnel may be inexperienced with the complications of Duchenne. In the case of an emergency room visit, it’s important that you are prepared to share your child’s medical information and restrictions. Keeping your medication information card up to date can help you and any of your child’s other caregivers stay prepared.
Cardiologists will be able to monitor your child’s cardiac condition throughout your Duchenne journey. The American Academy of Pediatrics recommends children affected by Duchenne begin seeing a cardiologist as soon as possible following their diagnosis.
An endocrinologist may be able treat concerns related to your child’s slowed development and delayed puberty.
Digestive System Specialist
Your child may experience symptoms related to the digestive system as a result of his Duchenne, including severe constipation and gastroesophageal reflux (when the tube to the stomach does not close properly and acid rises back up from the stomach, causing heartburn). A gastroenterologist may be able to help your child manage these symptoms.
Nervous System Specialist
Neurologists are interested in the diagnosis and progression of neuromuscular diseases, such as Duchenne. Neurologists may be able to prescribe your child medications, such as corticosteroids, that help manage the symptoms associated with Duchenne.
Bones and Joints Specialist
Duchenne doesn’t directly affect a patient’s bones but weakened bones may occur as a secondary result of damaged muscles. Your child affected by Duchenne may experience contracted muscles around joints and an orthopedist may be able to help relieve the stiffness with braces, exercise, and in some cases, surgery.
Brain Cognition and Emotion Specialist
Many patients affected by Duchenne may also experience learning disabilities. A psychologist can help diagnose and treat cognitive (ability to process information) or emotional problems associated with Duchenne.
The lungs are not directly affected by Duchenne since they are not muscles; however, because the lungs are supported by muscles that can be affected, it may benefit your child to see a pulmonologist.
A physical therapist can work with your child to emphasize his mobility, prevent contractures (hardening of joints), and scoliosis. In some cases, your child’s physical therapist may even be able to work with your child to strengthen his large muscle groups.
1. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopedic management. Lancet Neurol. 2018. doi:10.1016/ S1474-4422(18)30025-5. 2. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular,rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267. 3. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Lancet Neurol. 2018;17(5):445-455.