KANUMA is a treatment for patients with a rare disease known as Lysosomal Acid Lipase (LAL) deficiency. Patients with LAL deficiency have no or little LAL enzyme activity. This results in a build-up of fats within the cells of various tissues that can lead to liver, heart, and blood vessel disease and other complications.
LAL deficiency has two forms. The first type (referred to as Wolman disease) is seen in infants (around 2 to 4 months of age) and is often fatal in the first year of life. The second form (referred to as cholesteryl ester storage disease, or CESD) is milder with an onset in early childhood or later in life. Life expectancy of patients with CESD depends on the severity of the disease and complications from the disease.
Drug Trials Snapshots provide information about who participated in clinical trials that supported the FDA approval of new drugs. The information provided in these Snapshots also highlights whether there were any differences in the benefits and side effects among sex, race and age groups.
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