BACKGROUND: Volanesorsen, an investigational inhibitor of apoC-III synthesis, 
significantly reduced triglyceride levels in clinical trials in patients with 
familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized 
by marked chylomicronemia leading to a spectrum of symptoms, including recurrent 
abdominal pain and episodes of potentially fatal acute pancreatitis (AP).
OBJECTIVE: To determine the effect of volanesorsen on burden of disease on 
patients with FCS Methods: ReFOCUS was a retrospective global web-based survey 
open to patients with FCS who received volanesorsen for ≥3 months in an 
open-label extension study. The survey included questions about patients' 
experiences before and after volanesorsen treatment.
RESULTS: Twenty-two respondents had received volanesorsen for a median of 
222 days. Volanesorsen significantly reduced the number of symptoms per patient 
across physical, emotional, and cognitive domains. Significant reductions from 
baseline were reported for steatorrhea, pancreatic pain, and constant worry 
about an attack of pain/AP. Respondents reported that volanesorsen improved 
overall management of symptoms and reduced interference of FCS with work/school 
responsibilities. Reductions in the negative impact of FCS on personal, social, 
and professional life were also reported.
CONCLUSIONS: Treatment with volanesorsen has the potential to reduce disease 
burden in patients with FCS through modulation of multiple symptom domains.