Hathout Yetrib, Seol Haeri, Han Meng Hsuan J, Zhang Aiping, Brown Kristy J, Hoffman Eric P
Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
Clin Proteomics. 2016 Apr 5;13:9. doi: 10.1186/s12014-016-9109-x. eCollection 2016.
Assessments of disease progression and response to therapies in Duchenne muscular dystrophy (DMD) patients remain challenging. Current DMD patient assessments include complex physical tests and invasive procedures such as muscle biopsies, which are not suitable for young children. Defining alternative, less invasive and objective outcome measures to assess disease progression and response to therapy will aid drug development and clinical trials in DMD. In this review we highlight advances in development of non-invasive blood circulating biomarkers as a means to assess disease progression and response to therapies in DMD.
评估杜氏肌营养不良症(DMD)患者的疾病进展和对治疗的反应仍然具有挑战性。目前对DMD患者的评估包括复杂的身体测试和侵入性程序,如肌肉活检,而这些并不适用于幼儿。定义替代性的、侵入性较小且客观的结果指标来评估疾病进展和对治疗的反应,将有助于DMD的药物开发和临床试验。在这篇综述中,我们重点介绍了非侵入性血液循环生物标志物开发方面的进展,作为评估DMD疾病进展和对治疗反应的一种手段。