Muscular Dystrophy (Myolysis)

Kas Erimesi (Musküler Distrofi)

With stem cell therapy, the progression of Myolysis can be slowed down and regressed, and even stopped completely. The success rate of the treatment is proportional to the patient's age, the duration of the disease and the patient's condition.

  • The treatment of MYOLYSIS with stem cells is recognized as a new method that scientists are working on.
  • Stem cells have the ability to turn into muscle cells when they touch damaged muscle cells, and that being the case, they are used in the treatment of myolysis.
  • The number of cells to be administered is determined according to the age and weight of the patient. Treatment is carried out using mesenchymal stem cells (derived from the patient's own adipose tissue or bone marrow) or fetal stem cells. The decision is made according to the patient's condition. It is performed in 3 sessions 45 days apart or in 3 consecutive days.
  • A success rate of 88% was achieved in areas such as general improvement in quality of life of patients with different degrees of disease, regression of the disease and strengthening of the immune system.

What is Myolysis Disease?

Duchenne Muscular Dystrophy (DMD) is the most common muscle disease seen among boys. In this disease, a basic protein that is essential for muscle function is missing. In the absence of this protein, the muscles gradually weaken and the muscle tissue is replaced by adipose tissue.

Myolysis is caused by shorter-than-normal making of the Dystrophin protein, which is a muscle protein, as a result of a disorder in the gene programming it. Males have XY and females have XX chromosomes. There is X-linked inheritance because the chromosome carrying Dystrophin is the X chromosome. Since males have only one X chromosome, the disease is only seen in males. Rarely, it may occur due to mutations taking place while the baby is in the mother's womb.

In some children, parents may notice the swollen and hard appearance on calf muscles and consult a doctor. It is a genetic disease that can be distinguished by progressive damage of skeletal muscles, causing muscle weakness and atrophy.

It first involves the region around the shoulders, the hip girdle and thighs. The course of the disease is slow, and these patients can sometimes walk up to their 30s. Sometimes the heart muscle may also be involved.

What are the Symptoms of Myolysis?

  • It is experienced as getting tired too quickly, and as difficulty in carrying out activities such as getting up from the floor, climbing uphill and climbing stairs. It is usually diagnosed between the ages of 2-5. Some children fall behind compared to their peers at every stage of growth after birth, while others grow up with similar characteristics to their peers.
  • Very mild symptoms are usually observed during childhood. Learning to walk may be delayed. In the course of time, muscular weakness, difficulty in standing up, walking fast and climbing stairs, frequent falls, waddling gait, muscle aches and cramps are observed.
  • The weakness of the muscles around the hips and shoulders gradually progresses and the abdominal and back muscles weaken as well, and thus, they start walking sticking their abdomens forward. With the progression of weakness in the leg muscles, they start falling frequently, have difficulty in getting up and climbing stairs and begin to use wheelchairs between the ages of 9-11 upon losing their ability to walk. After this stage, the arm muscles gradually weaken as well and the person will have difficulty in using his/her arms.

Causes of Myolysis

The disease was either transmitted from the previous generation, or this gene was incorrectly formed while the baby was still in mother's womb. The result is the same in both cases. The sick gene is on the X chromosome, one of the gender chromosomes, which means that myolysis is a congenital and genetic disease.

Each person has 23 pairs of chromosomes and one of these chromosome pairs comes from the mother and the other from the father. Our genes that determine our genetic structure are also found on these chromosomes. A person carrying a genetic disease gene is more likely to have a close relative with the same disease gene than people who are unrelated. This means that the probability of passing on the gene incorrectly encoding the dystrophine to the infant also increases. Therefore, the incidence of the disease in children born as a result of consanguineous marriage is 2 times more than other infants.

Treatment of Myolysis

There is no known drug treatment for this disease at the moment, and there is no drug discovered which can completely eliminate the disease. In countries where research has been done on this subject, methods that can replace or work as dystrophin in muscle cells are being investigated.

Treatment of Myolysis with Stem Cells

The success rate of the treatment is proportional to the patient's age, the duration of the disease and the patient's condition. Stem cells have the ability to turn into muscle cells when they touch damaged muscle cells, and that being the case, they are used in the treatment of myolysis. However, because this disease is caused by a genetic disorder, the patient can be administered with genetically intact stem cells usually obtained from fetus.

On the other hand, if it is ensured that the stem cells obtained from the patient are not affected by the disease, the patient's own stem cells can also be used. In this way, they may slow the progression of the disease, completely stop it and make it regress. If the disease has severely progressed, treatment may need to be applied more than once. This disease can be treated at our GenCell Stem Cell Treatment Center in Kiev, Ukraine.

Frequently Asked Questions

Can Myolysis be detected before the child is born?

Pre-pregnancy genetic diagnosis is very important so that those who have been determined to be carriers as a result of tests performed have healthy children. However, it should be kept in mind that mutations that may occur in the mother's womb may also cause the disease. In addition, the disease can be detected by prenatal diagnostic methods such as such as amniocentesis. In this case, the family should take a decision regarding the continuation of pregnancy.

What are the considerations in Myolysis Disease?

It is up to the family to slow down the progression of this disease to a little extent, and the child's active efforts are also required. Daily exercises under the supervision of physiotherapists are very effective. Exercise therapy should be planned specific to the patient according to the degree of muscle weakness.

Scientific Studies:

  1. Périé, S., Trollet, C., Mouly, V., Vanneaux, V., Mamchaoui, K., Bouazza, B., … & Butler-Browne, G. (2014). Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study. Molecular Therapy, 22(1), 219-225.
  2. Ichim, T. E., Alexandrescu, D. T., Solano, F., Lara, F., Campion, R. D. N., Paris, E., … & Marleau, A. M. (2010). Mesenchymal stem cells as anti-inflammatories: implications for treatment of Duchenne muscular dystrophy. Cellular immunology, 260(2), 75-82.
  3. Maffioletti, S. M., Noviello, M., English, K., & Tedesco, F. S. (2014). Stem cell transplantation for muscular dystrophy: the challenge of immune response. BioMed research international, 2014.

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