Among the LSD-affected individuals, all individuals with acid lipase deficiency and 86%, 60%, and 58% of individuals with mucolipidosis II/III, Niemann-Pick (A/B) disease, and Gaucher disease, respectively, had total acid [alpha]-glucosidase concentrations higher than the 95th percentile of the control group (Table 1).
Synageva) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced that the first patient initiated treatment in the ARISE trial (Acid Lipase Replacement Investigating Safety and Efficacy), a global, Phase 3, randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with late onset lysosomal acid lipase deficiency (LAL Deficiency).
In addition, Alexion has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia and lysosomal acid lipase deficiency (LAL-D).
The drug, which would have been used to treat infants, children and adults suffering from lysosomal acid lipase deficiency (LAL-D), was rejected due to its high price and uncertainty surrounding its long term benefits.
Synageva) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Hepatology.