Muscular dystrophies are a category of disorders that gradually weaken and stiffen muscles. The most prevalent form is Duchenne muscular dystrophy (DMD). It's due to faults in a gene that regulates how the body maintains muscular health.
Almost majority cases of the illness involve boys, and symptoms generally appear in early childhood. Standing up, walking, and climbing stairs are difficult for children with
Duchenne muscular dystrophy. Many people eventually require the use of wheelchairs to go about. They may also suffer from heart and lung issues.
The outlook for persons with DMD is brighter than it has ever been, despite the fact that there is no treatment. Children with the illness used to seldom live into their adolescence. They now live well into their 30s, and occasionally into their 40s and 50s. There are treatments that can help with symptoms, and researchers are always seeking for new ones.
Symptoms- If your child has DMD, you'll likely notice the first symptoms before they reach the age of six.
- Leg muscles are generally the first to be damaged, therefore they'll start walking considerably later than normal youngsters their age.
- They may fall down frequently once they can walk and have difficulty ascending stairs or getting up from the floor.
- They may also begin to waddle or walk on their toes after a few years.
Treatment- Although there is no cure for DMD, there are medications and other treatments that can help your child's symptoms, safeguard their muscles, and maintain their heart and lungs in good working order
- Eteplirsen (Exondys 51): has been approved to treat individuals with a specific mutation of the gene that leads to DMD. It is an injectable medicine that aids in the healing process. Balance issues and vomiting are the most prevalent adverse effects. Despite the fact that the medication boosts dystrophin synthesis, which would suggest improved muscular function, this has yet to be demonstrated.
- Prednisone: Steroids such as prednisone slow muscle damage. Children who take this medication can walk for 2 to 5 years longer than those who do not. The medicines may also improve the function of your child's heart and lungs.
- Deflazacort (Emflaza): The oral corticosteroid deflazacort (Emflaza) was authorised by the FDA in 2017 to treat DMD, making it the first corticosteroid to be licenced for the disease. Deflazacort has been shown to assist patients preserve their muscular strength as well as their capacity to walk. Puffiness, increased hunger, and weight gain are all common adverse effects.
Children with DMD have lower bone density and are more likely to break specific bones, such as the hips and spine. Many people with this condition may experience mild to severe non-progressive intellectual impairment and learning problems.
Comments
Post a Comment