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目的观察干细胞移植治疗进行性肌营养不良症患儿的临床疗效,从而探索治疗进行性肌营养不良症的有效方法。方法观察10例进行性肌营养不良症患儿接受干细胞移植前后的生活能力及血清酶学改变,进行疗效评估。结果干细胞移植治疗可改善进行性肌营养不良患儿的生活能力,血清酶学下降。结论干细胞移植是治疗进行性肌营养不良症的一种有效手段,值得进一步研究及推广,在提高生活质量及血清降低酶学改变上可证实。
Objective To observe the clinical efficacy of stem cell transplantation in treatment of patients with progressive muscular dystrophy and to explore effective methods for the treatment of progressive muscular dystrophy. Methods Ten patients with progressive muscular dystrophy were evaluated for their viability and serum enzyme changes before and after stem cell transplantation. Results Stem cell transplantation improved the viability of children with progressive muscular dystrophy and decreased serum enzymology. Conclusion Stem cell transplantation is an effective method for the treatment of progressive muscular dystrophy. It is worth further study and dissemination. It can be confirmed by improving the quality of life and reducing serum enzymatic changes.