The global Pompe Disease market size reached USD 1,386.09 Million in 2021 and is expected to register a revenue CAGR of 3.9% during the forecast period, according to a latest analysis by Emergen Research. Significant variables influencing market revenue growth include the increased prevalence of Pompe disease, the number of clinical studies and trials in the area of treatments based on Pompe disease, and the launch of novel and sophisticated screening platforms for early diagnosis.

The heart and skeletal muscles are both impacted by the inherited, frequently deadly condition known as Pompe disease. The National Institute of Neurological Disorders and Stroke estimates that it occurs in one out of every 40,000 newborns. It is brought on by changes in a gene that codes for an enzyme called acid alpha-glucosidase (GAA).

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Numerous researchers are working to better understand the initial signs and symptoms of newborn screening-based novel screening platforms for late-onset Pompe illness. The Muscular Dystrophy Association (MDA) reports that 20 states are now screening babies for Pompe Condition, the newest neuromuscular disease being added to the list of important genetic disorders that children in the United States can be tested for shortly after birth.

We are looking into the potential effects of administering the proper amount of ERT to individuals early on. In addition to routine newborn screening, targeted screening of those at risk can result in the early identification and treatment of Pompe disease patients, potentially improving clinical outcomes. Large-scale screening programmes are developing, testing, and validating other cutting-edge and innovative diagnostic techniques, such as enzyme activity evaluation utilising a tandem-mass-spectrometry-based assay for Pompe disease.

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The research report offers in-depth insights into company profiles along with their production values, production capacity, product portfolio, strategic plans such as mergers and acquisitions, joint ventures, collaborations, product launches and brand promotions, government and corporate deals, among others. The report, additionally, offers a comprehensive SWOT analysis and Porter’s Five Forces analysis to offer a better understanding of the competitive landscape of the industry.

Emergen Research has segmented global Pompe Disease market on the basis of type, treatment type, end-use and region:

Type Outlook (Revenue, USD Million; 2019-2030)

Infantile-Onset Pompe Disease (IOPD)

Late-Onset Pompe Disease (LOPD)

Treatment Type Outlook (Revenue, USD Million; 2019-2030)

Enzyme Replacement Therapy (ERT)

Chaperone Therapy

Substrate Replacement Therapy (SRT)

Others

End-Use Outlook (Revenue, USD Million; 2019-2030)

Hospitals

Diagnostic Centers

Others

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The report also studies the key companies of the Pompe Disease market along with analysis of their business strategies, MA activities, investment plans, product portfolio, financial standing, gross profit margin, and production and manufacturing capacities.

Key players include

Sanofi S.A.,

Amicus Therapeutics, Inc.,

BioMarin Pharmaceutical,

Valerion Therapeutics,

Oxyrane,

Genzyme Corporation,

EpiVax, Inc.,

Bayer AG,

Audentes Therapeutics, and

Sangamo Therapeutics

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Regional Segmentation

North America (U.S., Canada)

Europe (U.K., Italy, Germany, France, Rest of EU)

Asia Pacific (India, Japan, China, South Korea, Australia, Rest of APAC)

Latin America (Chile, Brazil, Argentina, Rest of Latin America)

Middle East Africa (Saudi Arabia, U.A.E., South Africa, Rest of MEA)

Key Features of the Pompe Disease Market Report:

The report offers details about key drivers, restraints, opportunities, challenges, growth prospects, limitations, and threats

The report encompasses details about the key companies, product portfolio along with specifications, production valuation, and market shares

Evaluation of key current and emerging market trends and growth prospects

It also offers research-backed estimations for the forecast period of eight years, primarily to estimate the potential market growth

Brief overview of industry with regards to research and development, technological advancements, and product development

In-depth assessment of upstream raw materials, downstream buyers, demands, and current market scenario

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