MD treatment – Duchenne Muscular Dystrophy treatment

Muscular dystrophy (MD) refers to a number of inherited diseases characterized by progressive weakness and wasting of the muscles. The most common MD type is Duchenne's muscular dystrophy. Among others are Becker, limb girdle, congenital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and Emery-Dreifuss MD types.

Duchenne muscular dystrophy (DMD) is a hereditary X-linked disease developing as a result of mutation in the dystrophin gene that leads to deficiency of this protein. Dystrofin belongs to the large class of sarcolemmal proteins and glycoproteins. Dystrophin deficiency results in degeneration of cells, especially myocytes, and cells participating in muscle stimulation processes related to contractive activity. Dystrophin deficiency also leads to relaxation of the sarcolemma hitch, which, in turn, results in membrane rupture and launches the sequence of processes resulting in muscle fibre necrosis.

The types of MD differ by the location of the muscles affected, the rate of the disease progress, the age the first symptoms appear, etc. For example, the genes for Duchenne's that are X-linked express only in boys but are carried and passed on by women. Boys with Duchenne's MD are usually in a wheelchair by the age of 12 and rarely live past their teens.

There is no cure for muscular dystrophy but medications and therapy can slow the course of the disease. MD treatment with fetal stem cells proves to be one of the options leading not only to deceleration in muscles wasting but also to some regression of the main disease symptoms.

In the course of MD treatment with stem cells, pools of genetically healthy cells are created in the body of the patient; these cell pools are capable of producing dystrophin which is able to penetrate affected cells. In addition, in the processes of determination, differentiation, and morphogenesis, transplanted stem cells produce pools of specialized cells the patients body needs, such as muscular, nervous, endothelial, etc.

At present, Cell Therapy Center EmCell is following up more than 200 patients with Duchenne MD and other forms of muscular dystrophy. MD treatment is carried out at different stages of disease, at different age, at different grades of muscle atrophy, and at different grades of patients capacity for independent motion (bedridden, in a wheelchair, able to walk independently).

Our long-term experience of treating patients with muscular dystrophy allows for making the following conclusions:

Incredible but true: some of our patients in whom DMD diagnosis has been genetically confirmed in childhood at present have reached 29 years of age.

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MD treatment - Duchenne Muscular Dystrophy treatment