title: Clinical utility of serum biomarkers in Duchenne muscular dystrophy.
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.
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Yetrib
authors.initials: Y
authors.lastname: Hathout
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Haeri
authors.initials: H
authors.lastname: Seol
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Meng Hsuan J
authors.initials: MH
authors.lastname: Han
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Aiping
authors.initials: A
authors.lastname: Zhang
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Kristy J
authors.initials: KJ
authors.lastname: Brown
authors.affiliation: Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
authors.forename: Eric P
authors.initials: EP
authors.lastname: Hoffman
keywords: Biomarkers; Clinical outcomes; Duchenne muscular dystrophy; Mass spectrometry; Pharmacodynamic biomarkers; Proteins; SomaScan; Surrogate biomarkers; miRNA