Publications by authors named "Anne Katrine Andreasen"
Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
Eugen Mengel Marc C Patterson Rosalia M Da Riol Mireia Del Toro Federica Deodato Anne Katrine Andreasen
J Inherit Metab Dis
November 2021
Article Synopsis
- A clinical trial tested the drug arimoclomol in 50 patients aged 2-18, showing that it significantly slowed disease progression compared to a placebo, with a 65% reduction in annual worsening of symptoms.
- Despite some adverse events in both groups, arimoclomol was generally well tolerated, with fewer serious side effects compared to the placebo group.
Background: Fatigue in multiple sclerosis (MS) is a debilitating symptom and experienced by most patients. In recent studies investigating this phenomenon, the majority of patients had a relapsing-remitting disease course.
Methods: Patients with progressive MS participating in one of three treatment trials during a period from 2010 to 2014 were included.