The Lysosomal Acid Lipase Deficiency (LAL-D) Knowledge Centre has now launched on epgonline.org, intended for healthcare professionals involved in the treatment...
Alexion Pharmaceuticals has announced that the FDA has approved Kanuma (sebelipase alfa) for the treatment of patients of all ages...
Alexion Pharmaceuticals, Inc. announced that new interim data show that 80% of infants (8 out of 10) with rapidly progressive...
Alexion Pharmaceuticals, Inc. announced that the European Commission (EC) has approved Kanuma (sebelipase alfa) for long-term enzyme replacement therapy (ERT)...
Merck Inc., has announced that the company will not submit applications for regulatory approval for anacetrapib, its investigational cholesteryl ester...
Pompe disease is a rare, progressive neuromuscular disorder caused by deficiency of lysosomal acid α-glucosidase (GAA) and subsequent glycogen accumulation.
Alexion Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)...
Pompe disease is an inherited metabolic myopathy caused by deficiency of acid alpha-glucosidase (GAA), resulting in lysosomal glycogen accumulation.
Alexion Pharmaceuticals, Inc. announced that the combined interim data from two ongoing open-label studies VITAL study and CL-08 study show...