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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.
Technavio’s analysts forecast the global Pompe disease treatment market to grow at a CAGR of 11.85% during the period 2016-2020.
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:
• Americas
• APAC
• EMEA
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.
Key vendors
• Amicus Therapeutics
• Audentes Therapeutics
• Sanofi Genzyme
Other prominent vendors
• EpiVax
• Oxyrane
• Sangamo BioSciences
• Valerion Therapeutics
Market driver
• Special regulatory drug designations for orphan drugs
• For a full, detailed list, view our report
Market challenge
• Limited patient population
• For a full, detailed list, view our report
Market trend
• Emergence of immunotherapy for Pompe disease
• For a full, detailed list, view our report
Key questions answered in this report
• What will the market size be in 2020 and what will the growth rate be?
• What are the key market trends?
• What is driving this market?
• What are the challenges to market growth?
• Who are the key vendors in this market space?
• What are the market opportunities and threats faced by the key vendors?
• What are the strengths and weaknesses of the key vendors?
You can request one free hour of our analyst’s time when you purchase this market report. Details are provided within the report.
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.
Technavio’s analysts forecast the global Pompe disease treatment market to grow at a CAGR of 11.85% during the period 2016-2020.
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:
• Americas
• APAC
• EMEA
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.
Key vendors
• Amicus Therapeutics
• Audentes Therapeutics
• Sanofi Genzyme
Other prominent vendors
• EpiVax
• Oxyrane
• Sangamo BioSciences
• Valerion Therapeutics
Market driver
• Special regulatory drug designations for orphan drugs
• For a full, detailed list, view our report
Market challenge
• Limited patient population
• For a full, detailed list, view our report
Market trend
• Emergence of immunotherapy for Pompe disease
• For a full, detailed list, view our report
Key questions answered in this report
• What will the market size be in 2020 and what will the growth rate be?
• What are the key market trends?
• What is driving this market?
• What are the challenges to market growth?
• Who are the key vendors in this market space?
• What are the market opportunities and threats faced by the key vendors?
• What are the strengths and weaknesses of the key vendors?
You can request one free hour of our analyst’s time when you purchase this market report. Details are provided within the report.
Frequently Asked Questions
This market study covers the global and regional market with an in-depth analysis of the overall growth prospects in the market. Furthermore, it sheds light on the comprehensive competitive landscape of the global market. The report further offers a dashboard overview of leading companies encompassing their successful marketing strategies, market contribution, recent developments in both historic and present contexts.
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The report provides a detailed evaluation of the market by highlighting information on different aspects which include drivers, restraints, opportunities, and threats. This information can help stakeholders to make appropriate decisions before investing.
