Ann D'Hondt

TL;DR: A phase 3, randomised, double-blind, placebo-controlled, multicentre study in 76 hospitals and specialised clinics across North America, Latin America, Europe, and Asia to assess the efficacy and safety of eculizumab in patients with refractory myasthenia gravis.

TL;DR: This study provides Class II evidence that elevated concentrations of CSF pNfH and NfL can accurately identify patients with ALS and shows an added value as diagnostic biomarkers for ALS, whereas the prognostic value of pNFH andNfL warrants further investigation.

TL;DR: The 6MWD, but not the ActivLim score, is a sensitive outcome measure to follow up LOPD patients, and should even be further increased because of the long diagnostic delay.

TL;DR: This study shows that intracerebroventricular administration of 2 µg/day of vascular endothelial growth factor to patients with amyotrophic lateral sclerosis is feasible, results in detectable cerebrospinal fluid levels and is well tolerated in most patients.

TL;DR: Clinical and electrodiagnostic data at time of diagnosis provide additional prognostic information compared to other known prognostic factors, and diagnostic category according to Awaji and the extensiveness of LMN involvement contain the most additional value.