by Drs.Like Wu, Xiaojuan Wang, Bo Cheng, Susan Chu and Shengjie Liu
Wu Medical Center, Bejing, China
Progressive muscular dystrophy, PMD, is a group of hereditary, progressive skeletal muscle degenerative disease, pathology characteristic by skeletal muscle fiber degeneration and necrotic.
Major clinical manifestations are slowly progressing the muscular dystrophy and muscular weakness; Cardiac muscle atrophy and Skeletal muscle atrophy occurs in part types. Different gene defects result in a different onset time: early to fetus period or after adult stage.
According to different clinical manifestations or gene defects, clinical classifications are Congenital muscular dystrophy; Duchenne muscular dystrophy, DMD; Becker's muscular dystrophy, BMD; Limb-girdle muscular dystrophy and so on. DMD and BMD are the most common clinical types. From the name we can know that muscular dystrophy is progressive, but the speed of the diseases progress is different.
The most of PMD patients have poor prognosis, muscle paralysis and atrophy may cause patient to be disabled or/and have organ failure. Conventional medication treatment includes the symptomatic treatment, supportive treatment and rehabilitation training, which can maintain and prolong the time of independent ambulation of patient. But these treatments cannot stop the diseases progress because these conventional medication treatments can neither reduce the speed of muscle fiber damage degeneration, nor newly increase the number of muscle fiber.
Wu Stem Cells Medical Center research and application of Mesenchymal stem cells transplant; different from adult muscle fiber cell transplantation. The difficulty is you have to inject into every single dystrophic muscle fiber, treatment outcome of this kind of injection or even inject into a single muscle are not clear yet.
Wu Stem Cells Medical Center recently learned that mesenchymal stem cells can differentiate the muscle fiber cell, then these cells can secrete dystrophin which have a complement; they can effectively reduce the speed of damage in the muscle fiber, reduce creatine phosphokinase, stop progression of PMD and recover strength in parts of the muscle.