What Is The Best Medicine For Muscular Dystrophy?

Muscular dystrophy is a genetic disease and presently there is no cure for any form of muscular dystrophy.1 The best approach for the management of muscular dystrophy is to delay the progression of disease and to improve the quality of life of the patient.

What Is The Best Medicine For Muscular Dystrophy?

There is no best medicine for muscular dystrophy. It is a genetic disease involving the mutation of DMD gene. This gene is responsible for the synthesis of dystrophin.2 Due to the mutation in this gene, the muscle formed in the body and stiff and has very low stretching power. As the condition is genetic, there is presently no cure for any form of muscular dystrophy. The condition may be congenital or acquired and can be acquired in childhood or at the later stage of life. The treatment for muscular dystrophy is the gene therapy, which is currently in the clinical trial stage. The early results of the study conducted for the application of gene therapy in muscular dystrophy are promising. The patients who receive the gene therapy have restored some ability to synthesize dystrophin. The boy, suffering from muscular dystrophy, prior to surgery, was not able to climb and after surgery shown improvement. The approach in the gene therapy is to deliver the functional gene in the muscle cells so that the muscles so formed are stretchable and contains dystrophin. The best approach is the integrated approach wherein the following measures are used:

  1. Slowing The Progression Of Disease: It is the best approach wherein various drugs an exercise is used to slow down the progression of the disease.
  2. Managing The Complications: If the muscular dystrophy involves the muscles of the vital organs such as heart and lungs or diaphragm, the patient is at risk for developing life-threatening complications. Thus, various drugs are used to reduce complications.
  3. Devices: By using the devices, the quality of the life of the patient is improved and the patient becomes more independent. Various devices include braces and mobility aids.

Normal Treatment For Muscular Dystrophy

As the disease genetic and involves gene mutation, presently there is no cure for the disease. Gene therapy is under clinical trials that has provide some hope to the patients suffering from muscular dystrophy as it has partially restored the capacity of the cells to produce dystrophin. However, the general approach is to slow the progression of disease, manage the complications and improve the quality of life of the patients. Following are the measures applied:

Steroids: Steroids reduces the progression of disease by inhibiting the inflammatory mediators. These inflammatory mediators accelerate the destruction of muscles. Steroids for choice for muscular dystrophy are prednisone and deflazacort. But steroids, in chronic administration, have severe side effects. Research is on to identify the derivatives of steroids with lesser side effects.

Exercises: Physical therapy is provided to the patients suffering from muscular dystrophy. The exercise includes aerobic exercise and strength exercise.3 These exercises help in maintaining the muscle tone.

Anticonvulsants: Muscular dystrophy in the brain may lead to seizures. Thus, anticonvulsants are prescribed in such patients.

Immunosuppressants: These drugs are used to suppress the immune system. Suppression in immune system helps in delaying the progression of disease.

Mobility Aids: Muscular dystrophy results in the severe weakness in the muscles and the patient is not able to walk. Thus, various mobility aids are advised such as canes, wheelchairs and walkers.

Braces: Leg braces and spinal braces are used to make the patients more independent. They help in keeping the muscle relaxer and prevent muscle stiffness.

Cardiac Drugs: Cardiac complication is a life-threatening condition and can occur if the muscular dystrophy involves cardiac muscles. Various drugs are used to manage the condition such as beta blockers or calcium channel blockers.

Corrective Surgery: Sometimes, the patient of the muscular dystrophy requires corrective surgery to ease the symptoms of the disease. The general surgery related to muscular dystrophy includes foot surgery, insertion of feeding tube and the correction of curvature caused by scoliosis.

Conclusion

There is no cure for muscular dystrophy. Treatment options include the delay in progression of disease and managing the related complications. Therapy to cure the disease such as gene therapy is under trial.

References:

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