Muscular dystrophy is a condition occurs due to gene mutation.1 The gene is mutated due to which the dystrophin is not synthesized. This dystrophin is responsible for imparting stretching characteristics to the muscle. As it is genetic disease, there is no cure available for any form of muscular dystrophy; however the progression of the condition can be slowed.
Can You Be Cured Of Muscular Dystrophy?
The condition may be either congenital or acquired. If the muscular dystrophy is present by birth, it is congenital and if the condition of muscular dystrophy is developed later in the life, it is known as acquired. Further, the condition may be developed in childhood and also in adults. Presently there is no cure available to the patients suffering from muscular dystrophy. The therapy for treatment is under clinical trials and will take some time to hit the market. This is due to the fact that the disease is genetic and is due to mutation in the genes resulting in faulty gene structure. The gene mutation is spontaneous. The therapy which has shown some promising results is gene therapy.
In gene therapy, the approach is to deliver a copy of dystrophin gene to the muscle cells due to which the muscle formed are strong. Study have concluded that after the gene therapy, the patients began to produce at least some amount of dystrophin in their muscles. Presently, there is no treatment for muscular dystrophy and the best approach taken by the physicians is to slow the progression of the disease and reduce the symptoms and complications of the disease.
Slowing Of Muscular Dystrophy
Slowing of the muscular dystrophy and management of the symptoms and complications is the approach currently advised by the physician. Various measures are taken to reduce the progression of the disease and reduction in the symptoms of the disease.
Corticosteroids: Corticosteroids such as prednisone and deflazacort are used to reduce the action of inflammatory mediators against weak muscle cells. But in the long run they have their own side effects. Thus, various derivatives of these steroids are under trial which have lower side effects with comparable of high efficacy.
TAS-205: TAS-205 inhibits an enzyme that plays an important role in muscle damage. Thus, by inhibiting this enzyme, this compound reduces the progression of disease. This compound is currently under clinical trial.
Immunosuppressants: Cyclosporine also helps in slowing the progression of the disease.
Dietary Supplements: Muscle damage generates a large number of free radicals and the oxidative stress is increased. This initiates a cascade effect that will further damage the tissues. Dietary supplements such as antioxidants scavenge free radicals.
Exercises: Various exercises are advised to keep the muscles active and preventing them from stiffness. Exercise includes aerobic exercise for heart, strength exercise to improve muscle tone and stretching exercise to prevent muscle from stiffing.
Peptide Therapy: Peptides also slow the progression of muscular dystrophy.
Muscular Dystrophy Inheritance
Muscular dystrophy is the condition in which the protein responsible for imparting the stretching ability of the muscles is absent. This protein is known as dystrophin and the condition is due to gene mutation. The condition is inherited in the X-chromosome linked recessive pattern. It means that the gene is present on the X-chromosome. The condition inherited by male and female are different. Gene for the synthesis of the dystrophin protein are present on X-chromosome. Male contain XY chromosome, thus if the male inherits a faulty X-chromosome, there is no alternative source for the synthesis of dystrophin and the male will suffer from muscular dystrophy.
The female has the chromosome XX. Thus, if only a single X-chromosome is faulty, the gene on the other X-chromosome will synthesize dystrophin. In this condition the female will not suffer from this disease and can only be termed as carrier. In order to develop muscular dystrophy in females, both the X-chromosome should be mutated. In some muscular dystrophy disease, the mutation results in synthesis of another form of dystrophin which is smaller and partly functions like dystrophin.
There is no cure for any form of muscular dystrophy because it is a genetic disease. The progression of this disease can be slowed through corticosteroids, peptide therapy, exercises, dietary supplements and immunosuppressants.
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