Global Gaucher Disease Treatment Market: Trends, Share, Size, Growth, Opportunity and Forecast 2022-2028

 

A type of fat (lipid) called glucocerebroside cannot be broken down in people with Gaucher disease, an autosomal recessive inherited metabolic disorder. glucocerebrosidase is an enzyme used during body synthesis that breaks down and reprocesses glucocerebroside.

Gaucher Disease Treatment Market lessens symptoms and long-term harm to your body. Enzyme replacement therapy (ERT) and substrate reduction therapy are the two types of Gaucher disease treatments currently available (SRT).

A cure for Gaucher Disease Treatment Market must permanently undo the underlying genetic mutations that cause it, as inherited genetic disorders are caused by an altered sequence of genes found on chromosomes within each cell in your body (1). Currently, there is no treatment for Gaucher disease.

A genetic metabolic disorder called Gaucher disease prevents the breakdown of the lipid glucocerebroside. In the human body, an enzyme called glucocerebrosidase degrades and reprocesses glucocerebroside. Mutations in the GBA gene, which lead to very low levels of the glucocerebrosidase enzyme and slow glucocerebroside breakdown, are the cause of Gaucher disease.

A buildup of fatty substances in your organs, particularly your spleen and liver, is what causes Gaucher Disease Treatment Market. This causes these organs to grow, which may affect how well they function. Although there is no known cure for Gaucher disease, there are a number of treatments that can help manage symptoms, prevent permanent harm, and improve quality of life.

In enzyme replacement therapy, artificial enzymes are used to replace the damaged enzyme. The three ERT medications currently approved by the FDA are imiglucerase, velaglucerase alfa, and taliglucerase (Elelyso). Analgesics for bone pain, joint replacement surgery for chronic pain relief and function restoration, transfusions of blood products for severe anaemia and bleeding, anti-bone resorption medications, calcium, and vitamin D for osteoporosis, and analgesics for bone pain.

Key Players

Shire Human Genetics Therapies, Inc., Actelion Pharmaceuticals Ltd., Genzyme Corporation, and Pfizer, Inc. are some of the major companies active in the global market for treating Gaucher disease (acquired by Johnson & Johnson in June 2017). For the treatment of Gaucher disease, several drugs are being developed by companies like Lixte Biotechnology Holdings Inc, JCR Pharmaceuticals Co Ltd, Pharming Group NV, and Orphazyme ApS.