Pompe Disease Therapeutic MarketGLOBAL INDUSTRY INSIGHTS, TRENDS, OUTLOOK, AND OPPORTUNITY ANALYSIS, 2017-2025
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REPORT DESCRIPTION Pompe Disease Therapeutic Market – Overview Manufacturers are focusing on finding new solutions by expanding their pipeline to cater the demand for pompe disease therapeutics market. For instance, in September, 2017, Avrobio Inc. expanded their products pipeline with gene therapy to treat pompe disease by utilizing a proprietary lysosomal targeting sequence to deliver high levels of enzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics received orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of Pompe disease. This novel treatment paradigm consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance uptake, co-administered with AT2221, a pharmacological chaperone. 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. 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. Š Coherent market Insights. All Rights Reserved
REPORT DESCRIPTION
Pompe Disease Therapeutic Market Taxonomy: By Therapy Type: ●
Gene Therapy
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Enzyme Replacement Therapy (ERT)
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Chaperone-Advanced Replacement Therapy (CHART)
By Administrational Route: ●
Oral
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Parenteral
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REPORT DESCRIPTION Pompe Disease Therapeutic Market: Key Players ●
Amicus Therapeutics, Inc.
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Valerion Therapeutics.
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LLC.
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Sanofi S.A.
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Oxyrane.
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Sangamo Therapeutics, Inc.
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The high cost associated with the treatment and long duration of the treatment are the factors restraining growth of the pompe disease therapeutic market. For instance, according to a research paper published by Harvard School, in 2016, Lumizyme accounted for US$ 100,000 per year for children and US$ 300,000 per year for adult treatments.
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