openPR Logo
Press release

Pompe Disease Market Intelligence with Competitive Landscape 2024

10-16-2017 02:11 PM CET | Advertising, Media Consulting, Marketing Research

Press release from: Transparency Market Research

/ PR Agency: Transparency Market Research
Pompe Disease Market Intelligence with Competitive Landscape

Pompe disease is a rare, progressive, autosomal recessively inherited metabolic disorder, and is often a fatal muscular disease that affects one of the lysosomal enzymes. Pompe disease can also be called as a glycogen storage disease or acid alpha-glycosidase deficiency or GAA deficiency. It mainly occurs due to accumulation of glycogen in some tissues and organs, particularly muscles, leading to abnormal functioning. The inability of breaking the lysosomal glycogen lies in the mutation of a gene responsible for production of the enzyme acid alpha-glycosidase. This enzyme breaks glycogen into simpler form i.e. glucose but a mutation in the GAA gene prevents this activity. The stoppage of glycogen breakdown causes massive cellular dysfunction, with the marked involvement of the cardiac, smooth, and skeletal muscle. 

Request Sample Copy of the Report@

 https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=14102

 

Pompes disease is categorized on the basis of disease severity, disease progression and age, such as infantile-onset, non-classic infantile-onset and late onset. Within few months of birth, the infantile onset of Pompes disease starts showing its symptoms such as enlarged liver(hepatomegaly),muscle weakness(myopathy), heart defect, and poor muscle tone (hypotony).The infants affected with this disease face challenges of weight gain as well as hampered growth and breathing problems. The affected infant may have large protruding tongue. If untreated, the infant may die of heart failure in the first year of life itself. Non-Classic infantile-onset is usually seen at the age of 1 year. The disease symptoms include poor motor skills and a delayed muscle progress .They may show signs of hearing impairment and abnormally large heart. The muscle weakness may seriously lead to breathing problems. The Late-onset marks its symptoms by later stages of childhood, adolescent age or in adulthood. This form of late onset is a bit milder than the previous ones and doesn’t involve heart. The muscle weakness is generally observed in the legs, trunk and breathing muscles. The progress of the disorder in the muscles controlling breathing will ultimately lead to the respiratory failure.

Pompes disease can occur in various age groups and population across the world. The occurrence of the disease is 1 in 40,000 births in the U.S. In many cases, the people suffering from Pompes Disease in late onset of the disease remain undiagnosed. The symptoms of Pompes Disease are observed due to the complete or partial deficiency of the GAA enzyme. Diagnosis of Pompe disease can be performed by GAA activity assay. Following are the tests for the detection of Pompe disease:

Sequencing of the GAA gene
GAA activity assay in blood, cultured amniocytes, chorionic villi and cultured skin fibroblasts.
The marker, urinary hexose tetrasaccharide can be used to keep a track of patients suffering from Pompe disease
Cross Reactive Immunological Material (CRIM) analysis
Pompe disease can be treated by symptomatic, supportive and disease-specific ways. As the disease involves impairment of cardiac, respiratory and muscular systems, the treatment necessitates requirement of specialists with expertise in all these disorders. Cardiologists, dieticians, pediatricians, orthopedists may need to co-ordinate and design a well collaborated treatment plan for the patient. The US Food and Drug Administration has approved an enzyme replacement therapy- Myozyme® for the infantile onset of Pompe disease suffering patients of less than 8 years old. Whereas Lumizyme® is used for patients older than 8 years of age. The genetically engineered human GAA enzyme is administered intravenously every two weeks.

Increasing developments in the gene therapy and enzyme replacement therapies are driving factors for the growth of Pompe disease market. However, affordability of the expensive therapy treatments may pose a potential threat to these markets in developing markets. Major market players in the field of Pompe Disease therapeutics are Genzyme, BioMarin Pharmaceutical, and Amicus Therapeutics.There are some key vendors like Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.

Request TOC of the Report@

https://www.transparencymarketresearch.com/sample/sample.php?flag=T&rep_id=14102 

 

The report offers a comprehensive evaluation of the market. It does so via in-depth insights, understanding market evolution by tracking historical developments, and analyzing the present scenario and future projections based on optimistic and likely scenarios. Each research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology developments, types, applications, and the competitive landscape. 

About Us

Transparency Market Research (TMR) is a market intelligence company, providing global business information reports and services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insight for thousands of decision makers. TMR’s experienced team of Analysts, Researchers, and Consultants, use proprietary data sources and various tools and techniques to gather and analyze information.

Our data repository is continuously updated and revised by a team of research experts, so that it always reflects the latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports.

Contact Us

Transparency Market Research
90 State Street, Suite 700
Albany, NY 12207
Tel: +1-518-618-1030
USA – Canada Toll Free: 866-552-3453
Email: sales@transparencymarketresearch.com
Website: www.transparencymarketresearch.com/

This release was published on openPR.

Permanent link to this press release:

Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.

You can edit or delete your press release Pompe Disease Market Intelligence with Competitive Landscape 2024 here

News-ID: 775283 • Views: 252

More Releases from Transparency Market Research

Biomass Power Generation Market Likely to Experience a Tremendous Growth in Near …
𝐁𝐢𝐨𝐦𝐚𝐬𝐬 𝐏𝐨𝐰𝐞𝐫 𝐆𝐞𝐧𝐞𝐫𝐚𝐭𝐢𝐨𝐧 𝐌𝐚𝐫𝐤𝐞𝐭: 𝐈𝐧𝐭𝐫𝐨𝐝𝐮𝐜𝐭𝐢𝐨𝐧 The global biomass power generation market has been prognosticated by Transparency Market Research (TMR) to reveal a fragmented nature due to the presence of several players looking to establish their business in the industry. Some of the prominent companies in the market are Fourth Energy Ltd., Helius Energy Plc, Ameresco, Inc., The Babcock & Wilcox Company, and DONG Energy A/S. In order to set a stronger
Produced Water Treatment Market Release Latest Trends & Industry Vision by 2031
𝐏𝐫𝐨𝐝𝐮𝐜𝐞𝐝 𝐖𝐚𝐭𝐞𝐫 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐌𝐚𝐫𝐤𝐞𝐭: 𝐈𝐧𝐭𝐫𝐨𝐝𝐮𝐜𝐭𝐢𝐨𝐧 Transparency Market Research delivers key insights on the global produced water treatment market. In terms of revenue, the global produced water treatment market is estimated to expand at a CAGR of ~5% during the forecast period, owing to numerous factors regarding which TMR offers thorough insights and forecasts in its report on the global produced water treatment market. 𝐑𝐞𝐚𝐝 𝐫𝐞𝐩𝐨𝐫𝐭 𝐎𝐯𝐞𝐫𝐯𝐢𝐞𝐰 - https://www.transparencymarketresearch.com/produced-water-treatment-market.html The global produced water treatment
Pressure Sensitive Adhesives Market Analysis, Size, Share, Growth, Trends, and F …
𝐏𝐫𝐞𝐬𝐬𝐮𝐫𝐞 𝐒𝐞𝐧𝐬𝐢𝐭𝐢𝐯𝐞 𝐀𝐝𝐡𝐞𝐬𝐢𝐯𝐞𝐬 𝐌𝐚𝐫𝐤𝐞𝐭: 𝐈𝐧𝐭𝐫𝐨𝐝𝐮𝐜𝐭𝐢𝐨𝐧 Transparency Market Research delivers key insights on the global pressure sensitive adhesives market. In terms of revenue, the global pressure sensitive adhesives market is estimated to expand at a CAGR of 5.2% during the forecast period, owing to numerous factors, regarding which TMR offers thorough insights and forecast in its report on the global pressure sensitive adhesives market. 𝐑𝐞𝐚𝐝 𝐫𝐞𝐩𝐨𝐫𝐭 𝐎𝐯𝐞𝐫𝐯𝐢𝐞𝐰 -https://www.transparencymarketresearch.com/psa-market.html The global pressure sensitive adhesives market
Fuel Cell Market Know the Revenue and Profit-Sources of the Industry
𝐅𝐮𝐞𝐥 𝐂𝐞𝐥𝐥 𝐌𝐚𝐫𝐤𝐞𝐭: 𝐈𝐧𝐭𝐫𝐨𝐝𝐮𝐜𝐭𝐢𝐨𝐧 One of the major elements likely to drive the global fuel cell market is the growing need for alternative energy sources. Other variables that are projected to fuel the demand include lower impact on the environment and growth in private-public cooperation. Worldwide, governments are anticipated to assist these advancements by providing financial support for R&D activities and appropriate financial schemes. Development of a strong regulatory as well

All 5 Releases


More Releases for Pompe

Pompe Disease Therapeutic Market - Increasing prevalence of pompe disease in man …
Pompe Disease Therapeutic Market report is a exploration database spread across colorful runners with multiple tables and numbers in it. The exploration covers a precious source of perceptive information for business strategists. Pompe Disease Therapeutic Industry provides the overview with growth analysis and literal & futuristic cost, profit, demand and force data (as applicable). The exploration judges give an elegant description of the value chain and its distributor analysis. This comprehensive
Pompe Disease Drug Market Epidemiology 2030| Delveinsight
DelveInsight's Pompe Disease Market Insights, Epidemiology, and Market Forecast 2030 report delivers an in-depth understanding of the Pompe Disease, historical and forecasted epidemiology as well as the Pompe Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. The Pompe Disease market report provides current treatment practices, emerging drugs, Pompe Disease market share of the individual therapies, current and forecasted Pompe Disease market Size
Pompe Disease Therapeutic Market - Attractive Growth Opportunities 2017 - 2025
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
Pompe Disease Market Growth Analysis up to 2024
Pompe disease is a rare, progressive, autosomal recessively inherited metabolic disorder, and is often a fatal muscular disease that affects one of the lysosomal enzymes. Pompe disease can also be called as a glycogen storage disease or acid alpha-glycosidase deficiency or GAA deficiency. It mainly occurs due to accumulation of glycogen in some tissues and organs, particularly muscles, leading to abnormal functioning. The inability of breaking the lysosomal glycogen lies
Pompe Disease Market Size – Industry Share Report 2024
Pompe disease is a rare, progressive, autosomal recessively inherited metabolic disorder, and is often a fatal muscular disease that affects one of the lysosomal enzymes. Pompe disease can also be called as a glycogen storage disease or acid alpha-glycosidase deficiency or GAA deficiency. It mainly occurs due to accumulation of glycogen in some tissues and organs, particularly muscles, leading to abnormal functioning. The inability of breaking the lysosomal glycogen lies
Pompe Disease Market : Size, Status and Forecast 2016 - 2024
Pompe disease is a rare, progressive, autosomal recessively inherited metabolic disorder, and is often a fatal muscular disease that affects one of the lysosomal enzymes. Pompe disease can also be called as a glycogen storage disease or acid alpha-glycosidase deficiency or GAA deficiency. It mainly occurs due to accumulation of glycogen in some tissues and organs, particularly muscles, leading to abnormal functioning. The inability of breaking the lysosomal glycogen lies