Lysosomal acid lipase deficiency can lead to liver failure and early death. A recently published placebo-controlled trial shows that enzyme-replacement therapy improves plasma levels of lipids and ...

Researchers report results of a trial showing the efficacy of a new enzyme-replacement therapy for lysosomal acid lipase deficiency. In an accompanying editorial, an expert notes that this first-ever ...

Alexion announced that the Food and Drug Administration (FDA) has approved Kanuma (sebelipase alfa) for the treatment of patients with lysosomal acid lipase deficiency (LAL-D). The Food and Drug ...

Of the more than 50 known lysosomal storage diseases (LSDs)-rare inherited metabolic disorders-only seven can be treated with approved enzyme-replacement therapies. Lysosomal acid lipase deficiency ...

NEW HAVEN, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today that new interim data show that 80% of infants (8 out of 10) with rapidly progressive lysosomal acid ...

About Lysosomal Acid Lipase Deficiency (LAL-D) LAL-D is a genetic, chronic and progressive ultra-rare metabolic disease associated with devastating morbidities and premature mortality. In patients ...

A phase III trial of enzyme-replacement therapy for lysosomal acid lipase deficiency has shown the effectiveness and safety of sebelipase alfa, a recombinant lysosomal acid lipase. Lysosomal acid ...

The FDA announced its approval of Kanuma, an enzyme replacement therapy, for the treatment of adult and pediatric patients diagnosed with lysosomal acid lipase deficiency. Kanuma (sebelipase alfa, ...

BOSTON — Enzyme replacement therapy with sebelipase alfa can significantly reduce liver injury, liver fat, and dyslipidemia in patients with lysosomal acid lipase (LAL) deficiency, results from a ...

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) ...

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the European Commission (EC) has approved Kanuma™ (sebelipase alfa) for long-term enzyme replacement ...