| Literature DB >> 28628244 |
Tania F Gendron1,2, Lillian M Daughrity1, Michael G Heckman3, Nancy N Diehl3, Joanne Wuu4, Timothy M Miller5, Pau Pastor6,7, John Q Trojanowski8, Murray Grossman9, James D Berry10, William T Hu11, Antonia Ratti12,13, Michael Benatar4, Vincenzo Silani12,13, Jonathan D Glass11,14, Mary Kay Floeter15, Andreas Jeromin16, Kevin B Boylan17, Leonard Petrucelli1,2.
Abstract
As potential treatments for C9ORF72-associated amyotrophic lateral sclerosis (c9ALS) approach clinical trials, the identification of prognostic biomarkers for c9ALS becomes a priority. We show that levels of phosphorylated neurofilament heavy chain (pNFH) in cerebrospinal fluid (CSF) predict disease status and survival in c9ALS patients, and are largely stable over time. Moreover, c9ALS patients exhibit higher pNFH levels, more rapid disease progression, and shorter survival after disease onset than ALS patients without C9ORF72 expansions. These data support the use of CSF pNFH as a prognostic biomarker for clinical trials, which will increase the likelihood of successfully developing a treatment for c9ALS. Ann Neurol 2017;82:139-146.Entities:
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Year: 2017 PMID： 28628244 PMCID： PMC5676468 DOI： 10.1002/ana.24980
Source DB: PubMed Journal: Ann Neurol ISSN： 0364-5134 Impact factor: 10.422