Muscular Dystrophy Treatments Like Some Gene-Based Approaches Can Help In Reducing And Managing The Severe Symptoms Of The Disease
Duchenne Muscular Dystrophy (DMD) is a genetic X-linked
disorder and the most common progressive pediatric muscle disorder. Various
researches have been conducted on this disease to develop a treatment that is
safe and effective. Based on articles published in the English language between
2000 and 2014, current therapeutic options for this disease were assessed.
Granulocyte colony-stimulating factor (GCF) therapy and stem cell therapy were
considered to be contemporary therapies for this disease.
Muscular dystrophy is a group of genetic diseases that weaken muscles over time. It is characterized by a lack of dystrophin protein, which is necessary for muscle function. Without dystrophin, muscles cannot function normally, and symptoms include difficulty walking, swallowing, and muscle coordination. The symptoms can be mild or severe, and individuals can experience them at any age.
Muscular Dystrophy Treatment options vary, depending on the severity and type of muscular dystrophy. Some treatments involve corticosteroid medication, while others involve stem cell treatments. Genetic testing is also an option. A genetic test can help diagnose the disease and determine whether it will be passed on to the child. There is no cure for muscular dystrophy. However, physical therapy can help patients cope with their symptoms and maintain their strength and flexibility.
Treatments for muscular dystrophy can improve a person's quality of life and slow down or even reverse the progressive muscle deterioration associated with the disease. Current treatments are aimed at treating the underlying causes of the condition. One method of treatment is small molecules, which are chemical compounds that can easily enter cells and interact with their target. This target could be a protein or other molecules in the cell. Approximately 90% of therapeutics are small molecules.
Corticosteroid treatments are used for patients with muscular dystrophy. These drugs can prolong a person's life and slow down the deterioration of muscles. However, they come with their own side effects. Side effects can include weight gain, osteoporosis, and glaucoma. Furthermore, corticosteroid medications can cause fluid retention, which can lead to swelling of the legs. It is important to consult a physician when beginning a course of treatment.
Corticosteroids may help patients with muscular dystrophy walk longer and maintain greater muscle strength and functional abilities. They may be effective for up to two years. However, the side effects of corticosteroids may not be the same for all patients. Therefore, patients with muscular dystrophy should be informed about their potential side effects.
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