Pompe disease or also referred to as Glycogen storage disease type II is an autosomal recessive metabolic disorder that damages nerve cells and muscles throughout the body. It is typically caused due to the accumulation of glycogen in the lysosome, owing to deficiency of the lysosomal acid alpha-glucosidase enzyme.
Rising prevalence of Pompe disease is expected to drive growth of the global Pompe disease therapeutic market during the forecast period. According to the National Organization for Rare Disorders (NORD), around 1 in 40,000 births in the U.S. and the Netherlands are affected with Pompe disease. According to the same source, a recent study showed that the state of Missouri reported a higher prevalence of 1 in 5,463. Pompe disease can occur in various populations and ethnic groups across the globe. According to the Pompe Disease News, around 5,000 to 10,000 people have Pompe diseases worldwide. As a result of this, the demand for new therapeutic treatment options is increasing globally. Thus, these factors are expected to drive growth of the global Pompe disease therapeutic market during the forecast period. Furthermore, proactive initiatives taken by government authorities to develop and manufacture innovative products for the diagnosis and treatment of rare diseases are expected to propel the global Pompe disease therapeutic market growth over the forecast period.
However, the high cost associated with treatment is expected to hinder the global Pompe disease therapeutic market growth over the forecast period. Continuous research and development activities can present lucrative growth opportunities in the global Pompe disease therapeutic market in the near future. Among regions, North America is expected to witness significant growth in the global Pompe disease therapeutic market during the forecast period. This is owing to the presence of robust healthcare infrastructure across the region. Furthermore, Europe is expected to register a robust growth rate over the forecast period, owing to continuous research and development activities combined with proactive support from government authorities to improve the healthcare sector.
Key companies operating in the global Pompe disease therapeutic market are CENTOGENE AG, Amicus Therapeutics, Inc., AVROBIO, Inc., Sanofi, Valerion Therapeutics, Audentes Therapeutics, and Oxyrane.
For instance, in June 2020, Sanofi announced results of Phase III trial for its investigational enzyme replacement therapy (ERT) avalglucosidase alfa indicated for late-onset Pompe disease (LOPD) with clinically meaningful improvement.
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