Alexion has gained FDA approval for Kanuma, its treatment for the rare disease lysosomal acid lipase (LAL) deficiency. Alexion says Kanuma (sebelipase alfa) could eventually bring in more than $ ...

Meanwhile, AZ will also picks up three other drugs – Strensiq (asfotase alfa) for hypophosphatasia, Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency (LAL-D) and anticoagulant ...

Then, cholesteryl esters (CEs), the main form of cholesterol in lipoproteins, are hydrolyzed to free cholesterol (FC) at an acidic pH by lysosomal acid lipase (LAL), encoded by the LIPA (lipase A) ...

Some non-FH individuals had other inherited diseases, such as dominant dysbetalipoproteinemia, sitosterolemia and lysosomal acid lipase deficiency; alternatively, some individuals had poor living ...

[66] One can postulate that antiproteases might protect the mammary gland from local proteolysis (caused by leukocytic or lysosomal proteases ... and digestive lipase) act in the newborn to ...

Researchers have identified a potential treatment for Sandhoff and Tay-Sachs diseases -- two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and ...

Guidance on the use of capecitabine and tegafur with uracil for metastatic colorectal cancer TA61 27 May 2003 27 May 2003 Guidance on the use of drugs for early thrombolysis in the treatment of acute ...

Using single-molecule long-read sequencing of the CD36 gene, researchers identified 180 genetic variants, including 12 that alter the amino acid sequence. Among these, 4 premature termination ...

Current literature is restricted primarily by retrospective cohort studies, large database analyses, and single prospective randomized controlled study that explore the impact of donor characteristics ...