Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers.

Alexander J CammackNazem AtassiTheodore HymanLeonard H van den BergMatthew HarmsRobert H BalohRobert H BrownMichael A van EsJan H VeldinkBalint S de VriesJeffrey D RothsteinCaroline DrainJennifer Jockel-BalsarottiAmber MalcolmSonia BoodramAmber SalterNicholas WightmanHong YuAlexander V ShermanThomas J EsparzaDiane McKenna-YasekMargaret A OwegiCatherine Douthwrightnull nullAlexander McCampbellToby FergusonCarlos CruchagaMerit CudkowiczTimothy M Miller

Published in: Neurology (2019)

We present a thorough investigation of C9ALS natural history, providing the basis for C9ALS clinical trial design. We found that clinical features of this genetic subset are less variant than in singleton ALS. In addition, we identified important correlations of C9ALS patient pathologies with clinical and demographic data.

Keyphrases

amyotrophic lateral sclerosis
clinical trial
randomized controlled trial
genome wide
preterm birth
gene expression
big data
machine learning
weight loss
gestational age
artificial intelligence