Mucopolysaccharidosis (Morquio Syndrome), is a group of inherited disorder in which body becomes unable to breakdown the mucopolysaccharides. Accumulation of mucopolysaccharides in cells as well as connective tissues causes the malfunctioning of metabolic enzymes. This malfunctioning may lead to occurrence of variety of health problems. Mucopolysaccharidosis is autosomal dominant in most cases involving X-linked pattern of inheritance. The signs and symptoms of the disease varies by the type of mucopolysaccharidosis. Some of the other complications of the disease include affected physical appearance and malfunction of organ’s system functioning and its cognitive development in most cases.
The prevalence of mucopolysaccharidosis varies based on its types. According to the Orphanet, one person in a population of 100,000 is affected with mucopolysaccharidosis type I or Hurler syndrome. According to the Journal of Applied Genetics, a survey was conducted between 1970 to 2010, which found that the prevalence of mucopolysaccharidosis type III was 0.86 out of 100,000 live births. Additionally, the prevalence in Polish population was estimated to be 1.81 out of 100,000 individuals. The growth in the pipeline of mucopolysaccharidosis therapeutics is attributed to increasing technical advancements in molecular biology and genetics. Gene therapies, cell therapies, immunotherapies and microRNA are the first line of treatment of mucopolysaccharidosis. The funding from rare disease organizations is also one of the factors, which is expected to increase the collaboration between companies, associations and institutes for the development of therapies for the treatment of mucopolysaccharidosis.
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Some of the companies having a pipeline of Mucopolysaccharidosis therapeutics include, Ultragenyx Pharmaceutical Inc., Shire plc, BioMarin Pharmaceutical Inc., ArmaGen Inc., Genzyme Inc., JCR Pharmaceuticals Co., Ltd., Green Cross Corporation, Alexion Pharmaceuticals Inc., Concert Pharmaceuticals Inc.
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