Lysosomal Acid Lipase (LAL) Deficiency Treatment; Kanuma Is Approved to Treat LAL-D Patients of All Ages

 Lysosomal acid lipase (LAL) deficiency treatment is currently limited to control of cholesterol levels and to prevent premature atherosclerosis. In 2015, the United States Food and Drug Administration (USFDA) approved Alexion Pharmaceutical’s Kanuma, an innovative enzyme replacement therapy (ERT), the only therapy available for the lysosomal acid lipase (LAL) deficiency treatment in the U.S., Japan, and Europe Union. Kanuma (sebelipase alfa) is first approved therapy that helps treat the underlying cause of the disease.

Lysosomal Acid Lipase (LAL) Deficiency Treatment

 

Lysosomal acid lipase (LAL) deficiency is a rare genetic disease and cannot be cured by any medicine or drug. Lysosomal acid, also known as lysine, is an amino acid which is essential for the activity of many metabolic processes in the body. A deficiency of lysosomal acid results in laryngeal atresia or abnormal vocal cord development or laryngeal paralysis. The symptoms of lysosomal acid lipase deficiency are muscular weakness, drooling, depression, and facial warts. This disease is inherited in an autosomal dominant form and is very rare in males.

Lysosomal acid lipase (LAL) deficiency treatment may involve administration of an adequate diet rich in Lysine and fatty acids, and avoiding alcohol, caffeine, fatty foods, and tobacco. The balanced diet should consist of proteins, carbohydrates, fat, and essential amino acid. In order to increase the level of lysosomal acid in the body, it is beneficial to consume foods that are rich in this amino acid. Some of the best foods for providing lysosomal acid are eggs, meat, fish, dairy products, poultry, and legumes. Avoiding sugar, white flour, and white rice can greatly contribute towards the prevention of lysosomal acid lipase deficiency.

Lysosomal acid lipase (LAL) deficiency, also known as LAL deficiency or Wolman’s Disease, is an inherited, ultra-rare genetic disease affecting children as well as adults. Apart from the recently approved drug by FDA known as Kanuma, there is no approved lysosomal acid lipase (LAL) deficiency treatment available. Kanuma addresses the underlying cause of the disease by replacing the LAL enzyme that is deficient, and it is approved to treat lysosomal acid lipase deficiency patients of all ages.

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