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Pompe disease is a type of lysosomal storage disease (LSD) that is caused by mutations in the GAA gene. These mutations inhibit the action of GAA, which is an enzyme responsible for catabolizing glycogen. The market for Pompe disease in the US is estimated to show healthy growth during the forecast period and is positively influenced by factors like the reimbursement of treatment expenses by governments and vendors in some countries. These factors propel market growth during the forecast period and result in its healthy CAGR of more than 10% by 2019.
Based on the Pompe disease industry insights, eminent factors, such as the high-unmet medical needs of patients, to drive market growth during the forecast period. Drugs like Myozyme and Lumizyme fail to improve the neurological symptoms associated with the disease. This unavailability of a drug that is safe, effective, and less expensive is expected to bolster market growth during the forecast period.
The infantile-onset Pompe disease generally manifests itself within a few months after the baby is born and is characterized by symptoms like myopathy, hypotonia, hepatomegaly, and a defective heart. The adult-onset Pompe disease generally manifests itself during adolescence or adulthood. This disease is generally milder than its infantile-onset form and is characterized by symptoms like progressive muscle weakness in the legs and the trunk.
This market is highly lucrative and is characterized by the presence of a limited number of vendors. Out of all the vendors in this market, only Genzyme has produced an approved drug for the treatment of the disease. Vendors that have the capability to manufacture and market a drug that can effectively treat Pompe disease and also exhibit a high efficacy profile are expected to gain maximum traction in the market.
Key vendors in this market are
Other prominent vendors analyzed in this market study are Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.
Technavio’s market research analyst has estimated upcoming trends, such as the recent rise in public awareness, to spur market growth during the forecast period. The organization of various events and fundraising programs by the government and non-government organizations is expected to raise awareness and educate people about the disease, its diagnosis, and its treatment options. Organizations like the United Pompe Foundation, International Pompe Association, Canadian Association of Pompe, Acid Maltase Deficiency Association, and NORD help to spread awareness by promoting various online and offline events.
This report provides a number of factors contributing to the adoption, limitations, and opportunities of the pompe disease market in the US. It also offers an analysis of each factor and an estimation of the extent to which the factors are likely to impact the overall market growth.
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This is preliminary content. The table of contents is subject to change.
PART 01: Executive summary
PART 02: Scope of the report
PART 03: Market research methodology
PART 04: Introduction
PART 05: Market landscape
PART 06: Market segmentation by product
PART 07: Geographical segmentation
PART 08: Market drivers
PART 09: Impact of drivers
PART 10: Market challenges
PART 11: Impact of drivers and challenges
PART 12: Market trends
PART 13: Vendor landscape
PART 14: Key vendor analysis
PART 15: Appendix
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