Published On:August 3, 2017, 4:53 pm
Pompe disease is also referred Glycogen storage disease type II. It is an autosomal recessive metabolic disorder that damages nerve cells and muscle throughout the human body. It occurs due to the buildup of glycogen in the lysosome owing to lack of the lysosomal acid alpha-glucosidase enzyme. The accumulation of glycogen leads to gradual muscle failing throughout the body and strikes various tissues, especially in the liver, heart, the nervous system, and skeletal muscles. Pompe disease has an autosomal recessive inheritance pattern.
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The research team at Duke University has developed Myozyme (alglucosidase alfa, rhGAA) in 2006, which is the first treatment for patients with Pompe disease. The treatment was based on enzyme replacement therapy. Myozyme treatment extends overall and ventilator-free survival. Early diagnosis and treatment contribute to much better results. The treatment has some side effects which involve flushing, fever, skin rash, amplified heart rate and also shock; however, these are manageable.
The global Pompe disease treatment market is expected to have steady growth over the forecast period of 2017 to 2022 with moderate compound annual growth rate. The availability of compensation policies for the Pompe disease treatment is the major factor that will positively impact the growth of this market, as the treatment costs approximately USD 1 million to USD 3 million for a patient each year, causing difficulties for patients to persist with the treatment for prolonged period. However, financial support from various organizations and initiatives from the governments of countries about favorable guidelines for the refund of patients undergoing treatment will also boost the growth of the global market in the coming years. Additionally, advancements in preventing Immunoglobin (IgG) will propel the market industry further. The only restraining factor for the global Pompe disease treatment market is the high cost of treatment in the remote and rural areas where organizational or government support may not be available.
The key market players in the global Pompe disease treatment market are Sangamo Therapeutics, Inc., Sanofi Genzyme, Oxyrane, Amicus Therapeutics, Valerion Therapeutics, LLC, BioMarin Pharmaceutical Inc., EpiVax, Inc. and Audentes Therapeutics, Inc. According to product types, the market industry is split into; primarily split into Tablet, Injection and Other. By application, the global market is classified as; Infantile-Onset Pompe Disease and Adult-Onset Pompe Disease. Geographically, the global dental services market is segmented into regions which are; Southeast Asia, Asia-Pacific, North America, Europe, and rest of the world.
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