About Pompe Disease Treatment
Lysosomal storage diseases (LSDs) are a group of progressive, autosomal recessive, and hereditary disorders characterized by the accumulation of specific substrates and variable age of onset and clinical symptoms. Pompe disease, a type of LSD, is caused by mutations in the GAA gene. These mutations inhibit the mechanism of action (MOA) of GAA; it is an enzyme responsible for catabolizing glycogen, resulting in accumulation of glycogen to toxic levels in the lysosomes of the cells. The buildup of glycogen in certain tissues and organs, especially in the muscles, hinders the normal functions of the organs. It can be further divided as adult-onset and infantile-onset Pompe diseases. As the disease is an autosomal recessive hereditary disorder, both parents are carriers and pass one copy of the mutated gene to their offspring. It affects both women and men across all ethnicities and races and has a varied age of onset of symptoms. Till date, researchers have identified over 300 genetic mutation types that lead to disease manifestation. Though there is no known cure for Pompe disease, enzyme replacement therapy (ERT) and diet therapy have been effective in reducing its clinical manifestations.
Technavios analysts forecast the global Pompe disease treatment market to grow at a CAGR of 11.85% during the period 2016-2020.
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Global Pompe Disease Market 2016-2020
Report recognizes the following companies as the key players in the global Pompe disease treatment market: Amicus Therapeutics, Audentes Therapeutics, and Sanofi-Genzyme
Other Prominent Vendors in the market are: EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics
Commenting on the report, an analyst from Reports team said: Usually, a substantial amount of revenue is spent on spreading awareness of the drug and disease, after its launch. Also, the post-marketing response for the drug involves approaching all the diseased individuals. Therefore, the limited population of Pompe disease patients worldwide results in reduced expenditure on, advertising, marketing, and awareness programs.
According to the report, the US FDA’s OOPD aims to develop and evaluate biologics, drugs, and devices that effectively cure and diagnose rare diseases, and extends benefits to underlying companies. It also grants orphan status to biologics and drugs proven to be effective and safe in treating rare diseases. The Orphan Products Clinical Trials Grants Program funds the clinical research tests to check the efficacy and safety of drugs, medical devices, and biologics.
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Further, the report states that various events and fundraising programs are being organized by government and non-government organizations to raise awareness and educate people about the Pompe disease and its diagnosis and treatment options resulting in social and economic benefits and improvement in patients’ quality of life. Organizations such as the United Pompe Foundation, International Pompe Association, Canadian Association of Pompe, Acid Maltase Deficiency Association, and National Organization for Rare Disorders (NORD) are involved in promoting various events online and offline to spread awareness.
The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.
Covered in this report
The report covers the present scenario and the growth prospects of the global Pompe disease treatment market for 2016-2020. To calculate the market size, the report considers the revenue generated from the sales of the drugs available in the market and the drug candidates that are in various phases of development for the treatment of Pompe disease.
The market is divided into the following segments based on geography:
Technavio’s report, Global Pompe Disease Treatment Market 2016-2020, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.
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