CNMC0705: A DOUBLE-BLINDED RANDOMIZED PLACEBO CONTROLLED STUDY OF DAILY

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 杜氏肌营养不良症（DMD）是一种进行性骨骼肌疾病，由于X连锁肌营养不良蛋白基因的遗传突变导致肌营养不良蛋白缺失。肌营养不良蛋白的缺乏导致脆弱的肌肉膜，允许电解质，特别是钙的异常渗透性。细胞内钙的增加触发了一系列病理性事件，最终导致肌肉坏死和纤维化，从而阻碍了正常的肌肉再生。 DMD的病理生理学知识的增加为药物治疗打开了机会，目的是改变疾病过程和/或逆转肌肉变性。戊茶碱（PTX）是一种有效的抗炎和抗纤维化化合物，在运动的mdx小鼠中产生51%的力量增加。它在DMD中的疗效尚未得到证实。然而，DMD和其他肌营养不良症的初步研究令人鼓舞。DMD儿童谁是弱，但稳定或恶化，尽管所有已证实的治疗类固醇治疗可能会受益于增加PTX治疗。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Duchenne muscular dystrophy (DMD) is a progressive disease of skeletal muscle caused by the absence of dystrophin due to a genetic mutation in the X-linked dystrophin gene. The absence of dystrophin results in a fragile muscle membrane that permits an abnormal permeability to electrolytes, especially calcium. The increase in intracellular calcium triggers a pathological cascade of events that ultimately resultsin muscle necrosis and fibrosis which impedes normal muscle regeneration. The increase knowledge of the pathophysiology of DMD opens the opportunity for pharmacological treatment, with the purpose of altering the disease processs and or reverting the muscle degeneration. Pentoxifylline (PTX) is a potent anti-inflammatory and anti-fibrotic compound that produced 51% increase in strength in exercised mdx mice. Its efficacy in DMD is not proven. However preliminary studies in DMD and other muscular dystrophyies are encouraging. DMD children treated with steroid who are weak but stable or deteriorating despite all proven treatments may benefit from the addition of PTX treatment.