Pompe Disease Therapeutic Market Global Industry Insights, Analysis, Trends and Forecast, 2017-2025
Increasing government initiatives is expected to augment growth of the pompe disease therapeutic market
Increasing government initiatives is expected to augment growth of the pompe disease therapeutic market
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REPORT DESCRIPTION<br />
<strong>Pompe</strong> <strong>Disease</strong> <strong>Therapeutic</strong> <strong>Market</strong> – Overview<br />
Manufacturers are focusing on finding new solutions by exp<strong>and</strong>ing their pipeline to cater the dem<strong>and</strong> for<br />
pompe disease therapeutics market. For instance, in September, <strong>2017</strong>, Avrobio Inc. exp<strong>and</strong>ed their products<br />
pipeline with gene therapy to treat pompe disease by utilizing a proprietary lysosomal targeting sequence to<br />
deliver high levels of enzyme to lysosomes. Furthermore, in October <strong>2017</strong>, Amicus <strong>Therapeutic</strong>s received<br />
orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of <strong>Pompe</strong> disease. This<br />
novel treatment paradigm consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA)<br />
enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance uptake,<br />
co-administered with AT2221, a pharmacological chaperone.<br />
Increasing prevalence of pompe disease in many economies is expected to have a lucrative opportunities for<br />
pharmaceutical manufacturers to come with a tailored solution for diagnosis <strong>and</strong> treatment of disease.<br />
<strong>Pompe</strong> disease is an inherited Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid<br />
named alpha-glucosidase (GAA). This causes the glycogen to accumulate in different kinds of tissues,<br />
primarily into smooth muscles, skeletal muscles <strong>and</strong> cardiac muscle. It leads to muscle weakness, respiratory<br />
problems, <strong>and</strong> may also affects the liver <strong>and</strong> heart.<br />
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