Considering the public health problems, government is taking continuous initiatives in promoting and advancing the development of innovative products for the prevention and diagnosis or treatment of rare diseases or conditions such as Pompe disease. For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards from 92 grant applications and provided funding and support to around 67 ongoing clinical study projects related to rare disease. Furthermore, initiatives from the governments of economies such as Sweden, the Netherlands, and South Korea that focus on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT) are also expected to boost growth of the pompe disease therapeutic market in near future.
Pompe disease is an inherited Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid named alpha-glucosidase (GAA). This causes the glycogen to accumulate in different kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac muscle. It leads to muscle weakness, respiratory problems, and may also affects the liver and heart. Pompe disease is inherited as an autosomal recessive genetic trait. There are three major types of pompe disease, which differ in severity and appear at different age, these types are known as classic infantile-onset, which starts within a few months of birth, non-classic infantile-onset Pompe disease- usually appears by age 1, and late-onset type of Pompe disease that may shows the symptoms later in childhood, adolescence, or adulthood. Pompe disease therapeutic market can exhibit immense growth potential due to presence of limited number of manufacturers, which can significantly enhance and expand their business potential and scope in this market.
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Increasing prevalence of pompe disease in many economies is expected to have a lucrative opportunities for pharmaceutical manufacturers to come with a tailored solution for diagnosis and treatment of disease. For instance, according to United Pompe Foundation (UPF), 2017, the estimated frequency occurrence of Pompe disease may vary among different ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1 in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000 births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000 suffer from pompe disease. Gene therapy and enzyme replacement therapy may be highly effective at reducing clinical manifestations of this rare disease.
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