Clinical utility of serum biomarkers in Duchenne muscular dystrophy

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 alternati...

Descripción completa

Detalles Bibliográficos
Autores principales: Hathout, Yetrib, Seol, Haeri, Han, Meng Hsuan J., Zhang, Aiping, Brown, Kristy J., Hoffman, Eric P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2016
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4820909/
https://www.ncbi.nlm.nih.gov/pubmed/27051355
http://dx.doi.org/10.1186/s12014-016-9109-x
Descripción
Sumario: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. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s12014-016-9109-x) contains supplementary material, which is available to authorized users.

Ejemplares similares