Synageva expands clinical trial in infants with LAL deficiency

The company said there are no therapies for patients with LAL Deficiency.

However, SBC-102 can possibly be a disease modifying long-term enzyme replacement therapy for patients including infants, children, adolescents and adults suffering with LAL Deficiency.

LAL Deficiency results in liver and spleen enlargement, fibrosis, cirrhosis and liver failure, if it occurs after infancy.

Investigator for clinical trials in both the early and late onset populations Dr. Vassili Valayannopoulos said that with the investigational therapy, diagnosis should be considered in children and adults with unexplained liver dysfunction and an abnormal lipid profile.