Clinical utility of serum biomarkers in Duchenne muscular dystrophy

Clinical Proteomics

Kristy J. Brown¹  Meng Hsuan J. Han¹  Eric P. Hoffman¹  Yetrib Hathout¹  Haeri Seol¹  Aiping Zhang¹ 

[1] Center for Genetic Medicine, Children’s National Healthy System, Washington, USACenter for Genetic Medicine, Children’s National Healthy System, Washington, USACenter for Genetic Medicine, Children’s National Healthy System, Washington, USA

关键词: Duchenne muscular dystrophy;    Biomarkers;    miRNA;    Proteins;    Pharmacodynamic biomarkers;    Surrogate biomarkers;    Clinical outcomes;    Mass spectrometry;    SomaScan;   

DOI  :  10.1186/s12014-016-9109-x

来源: Humana Press Inc

【 摘 要 】

Abstract

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.

【 授权许可】

Unknown