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What is Duchenne Muscular Dystrophy

Duchenne muscular dystrophy treatment in UCTCDuchenne muscular dystrophy is a disorder that is caused by lack of dystrophin, which is a protein in the muscles. When person is affected with this disease, that brings to muscle weakness. The disease is progressing rather fast. Weakness starts in lower limbs as a rule and spreads rapidly.

Who is affected

This serious condition is inherited, but there are cases when people who do not have patients with this diagnosis in the family are affected. The condition occurs in boys for the most part because of the specific character the gene is inherited.

Development of condition

The condition starts developing before the boy is 6 years old as a rule and the first symptoms can be revealed in infancy period. When motor functions are damaged, the person is not able to stand or sit on his own. The weakness progresses in legs and pelvic muscles therefore the patients has problems when walking and climbing stairs. Waddling gait can be one of the symptoms of Duchenne musclular dystrophy. After lower limbs are affected the diseases climbs up and reaches other areas, such as arms, neck and etc. The diminishing muscle strength continues until 10 years old and patients need braces support to be able to walk independently. Then 2 years later they are not able to walk and need wheelchair. The condition deforms the skeleton and spine. Weakness in the muscles brings to problems with breathing and causes intellectual impairment. Patients usually are not able to survive long and do not reach the age of 30. Lethal outcome occurs usually as a result of breathing complications or cardiomyopathy.

Symptoms of Duchenne muscular dystrophy

The patients experiences the following symptoms:

  • Tiredness
  • Learning problems
  • Weakness in the muscles that entail to motor malfunctions, problems with gait and raising from lying position
  • Falls
  • Deteriorated weakness

Examination and tests

The disease can be diagnosed differently:

  • Via muscle biopsy, when level of dystrophin is studied
  • Genetic testing on blood

It is usually possible to detect the condition using these methods in 95% of people diagnosed with Duchenne muscular dystrophy.

Treatment for Duchenne muscular dystrophy

When treatment is appointed the aim is to treat the symptoms. The devices for easier breathing may be required, especially at night time. Prednisone is appointed to enhance functions of patients with DMD.

When Prednisone is taken then ability to walk independently is preserved longer. For instance, patient without Prednisone can go on like this for a couple of years, while the other one, who gets Prednisone treatment course can make it up to 5 years. The patient should be ready for side effects that include weight gain, change of behavior pattern, high blood pressure and delayed development.

The doctor can also prescribe amino acids, creatine, fish oil, carnitine, vitamin E for maintenance therapy, but there are no clinical studies that prove positive effects of such treatments.

Stem cell therapy for patients with DMD

a) Research

Healthy transplants taken from mice were injected to mice with DMD. The transplantation was done after the dose of radiation. As a result of research it was proved that transplanted stem cells have potency to regenerate older damaged muscles. When the condition of mice was assessed 3 weeks later, it was seen that more stem cells were repopulated. This proved that even over time transplanted cells remain functional.

b) Use of stem cells therapy for people

Researchers’ conclusions seem to be promising but further research is still under way. Chronic muscle damage can be slowered. When treatment is focused on muscle fiber only, this is not enough. But when therapy replenishes the stem cell compartment, better results can be achieved.

At present stem cell treatment is organized at Unique Cell Treatment Clinic that are able to enhance the condition of the patient, prolong his life considerably and improve the patient’s quality of life. Specialists of UCTC can help you in treatment different diseases: ulcerative colitis, diabetes, anemia, cancer etc.