STEROID THERAPY IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

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， 因此可以在其他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。 前瞻性/随机 多机构：13个CINRG中心 受试者人群：确诊DMD的4-10岁非卧床男孩，未经糖皮质激素治疗。 受试者数量：140 治疗组（每组35例患者） 4-<7岁，每日泼尼松 4-<7岁，每周大剂量泼尼松 7+至10岁，每日泼尼松 7+至10岁，每周一次大剂量泼尼松 主要终点： 力量：上肢和下肢QMT评分的百分比变化。 副作用：治疗前后身高和体重/身高比变化的绝对平均值差异。 研究持续时间：12个28天治疗周期。 （约12个月）。 随访频率：第1个月和第3个月访视，然后每3个月访视一次。

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. Prospective/Randomized Multi-Institution: 13 CINRG centers Subject Population: Ambulant boys aged 4-10 years with established DMD diagnosis, naive to treatment with glucocorticoids. Number of Subjects: 140 Treatment groups (35 patients per group) 4-<7 years of age daily prednisone 4-<7 years of age high-dose weekly prednisone 7+ to 10 years of age daily prednisone 7+ to 10 years of age high-dose weekly prednisone Primary Endpoints: Strength: Percent change in QMT scores of upper and lower extremities. Side-Effects: Difference in absolute means of the pre and post treatment change in height and weight/height ratio. Duration of Study: twelve (12) 28-day treatment cycles. (Approximately 12 months). Frequency of Follow-Ups: Visits at month 1 and month 3, then every 3 months.