Sebelipase alfa is used to treat lysosomal acid lipase deficiency (LAL-D) and was evaluated in a study to assess its efficacy and safety in patients over a 144-week period.
Thirty-one patients received sebelipase alfa intravenously, with dose adjustments based on tolerance, and showed significant improvements in liver function, cholesterol levels, and overall health indicators over the study duration.
The treatment was generally well-tolerated, with mostly mild side effects, and led to positive outcomes in liver histopathology and lipid profiles.