Neurofilament as a potential biomarker for spinal muscular atrophy

Autor:	Eugenio Mercuri, Wildon Farwell, Darryl C. De Vivo, Eduardo F. Tizzano, Richard S. Finkel, John F. Staropoli, Christopher Stebbins, Thomas O. Crawford, Guolin Zhao, Maryam Oskoui, Charlotte J. Sumner, Marco Petrillo, Francesco Muntoni, Alexander McCampbell, Basil T. Darras, Stephanie Fradette, Monique M. Ryan
Jazyk:	angličtina
Rok vydání:	2019
Předmět:	Male 0301 basic medicine Treatment response medicine.medical_specialty Neurofilament Adolescent Intermediate Filaments Neurosciences. Biological psychiatry. Neuropsychiatry Gastroenterology law.invention Muscular Atrophy Spinal 03 medical and health sciences 0302 clinical medicine Double-Blind Method Randomized controlled trial law Internal medicine medicine Humans Child RC346-429 Research Articles business.industry General Neuroscience Spinal muscular atrophy medicine.disease SMA 030104 developmental biology Potential biomarkers Biomarker (medicine) Female Nusinersen Neurology (clinical) Neurology. Diseases of the nervous system business Biomarkers 030217 neurology & neurosurgery Research Article RC321-571
Zdroj:	Annals of Clinical and Translational Neurology, Vol 6, Iss 5, Pp 932-944 (2019) Annals of Clinical and Translational Neurology
ISSN:	2328-9503
Popis:	Objective To evaluate plasma phosphorylated neurofilament heavy chain (pNF‐H) as a biomarker in spinal muscular atrophy (SMA). Methods Levels of pNF‐H were measured using the ProteinSimple® platform in plasma samples from infants with SMA enrolled in ENDEAR (NCT02193074) and infants/children without neurological disease. Results Median pNF‐H plasma level was 167.0 pg/mL (7.46–7,030; n = 34) in children without SMA (aged 7 weeks–18 years) and was higher in those aged
Databáze:	OpenAIRE
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