@rpavaneijk.bsky.social & colleagues conducted a multi-year prospective cohort study of 97 patients with #ALS. Their findings: an #accelerometer -based measure is predictive of disease progression and related to patient-reported functioning: www.thelancet.com/journals/ebi...

Our study on the safety and tolerability of Trimetazidine in #ALS @braincomms.bsky.social Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study @rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social academic.oup.com/braincomms/a...

Dropout & attrition in #ALS #MND trials is a major challenge. We have created an #open-access calculator to estimate attrition rates for a given study duration, which can used to guide trial design and sample size calculations (tricals.shinyapps.io/Attrition/); the accompanying paper: rdcu.be/d3n36

New paper to reflect on the main challenges in phase 2 #ALS #MND trials. Key issues relate too short study durations, reliance on the wrong outcome measures, absence of biomarkers, statistical issues, over-interpretation of 'clinical trends' and miscommunication. academic.oup.com/brain/articl...