#Pompe Disease Treatment Market Forecast

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The global pompe disease treatment market is projected to witness significant growth during the forecast period. In U.S., projected occurrence are approximately 1 in 40,000 births. Males and females are equally affected by the diseases. Medical advancements that involves ongoing drug discovery, and government and non-government organizational initiatives are what that boosts the market growth.  

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Enzyme Replacement Therapy Market Outlook: Regional and Global Perspectives

The global enzyme replacement therapy market size is estimated to reach USD 17.26 billion by 2030, registering to grow at a CAGR of 9.0% from 2025 to 2030 according to a new report by Grand View Research, Inc. As per the Genetic and Rare Diseases Information Center or GARD, a rare disease is defined as a condition that affects lesser than 200,000 individuals. These disorders are also called orphan diseases due to the lack of support exhibited by pharmaceutical companies previously. Since the passing of the Orphan Drug Act of 1983 in the U.S., several large pharmaceutical and biopharmaceutical companies have invested heavily in the development of orphan drugs. Thus, rising awareness, increasing R&D investment, growth in the number of lysosomal storage and genetic disorders, and easy drug approval process are factors propelling the market growth during the forecast period.

According to data from the National Institutes of Health, it is estimated that over 25 million Americans suffer from rare diseases. These facts are indicative of the fact that although the number of these ailments is low, individuals suffering from these are large. Some of the most common lysosomal storage disorders that make use of enzyme replacement therapy are Gaucher, Fabry, Pompe, MPS, SCID, and Shwachman-Diamond Syndrome among others.

In the recent past, these disorders have been gaining significant attention, impacting growth positively. Various grants and acts are in place to accommodate drug makers to invest in the development of orphan drugs. Orphan Drug Designation Program, Rare Pediatric Disease Priority Review Voucher Program, Humanitarian Use Device (HUD) Program, and Orphan Products Clinical Trials Grants Program are programs that aid in the development of orphan drugs.

The rising use of enzyme replacement therapy in home care and infusion centers are gaining traction in the market. Specialized infusion centers such as these are anticipated to witness the highest CAGR during the forecast period. Hospitals held the largest segmental share owing to easy accessibility and ability to treat a wide range of ailments.  

Enzyme Replacement Therapy Market Report Highlights

Gaucher disease held the dominant position in the market and accounted for the largest revenue share of 32.6% in 2024, driven by the increasing prevalence and awareness of the condition, leading to more patients being diagnosed and treated.

Imiglucerase dominated the market and accounted for the largest revenue share of 20.2% in 2024, owing to the rising prevalence of Gaucher disease, which necessitates effective treatment options. In addition, as awareness and diagnosis rates improve, more patients are being identified, increasing demand for imiglucerase.

The parenteral route of administration dominated the market and accounted for the largest revenue share of 80.9% in 2024. This growth is attributed to its ability to facilitate rapid absorption and effective drug delivery. In addition, this method allows for intravenous infusions, which are essential for delivering large molecules such as enzymes directly into the bloodstream, ensuring immediate therapeutic effects

Infusion centers led the market and accounted for the largest revenue share of 40.8% in 2024, driven by their specialized focus on intravenous treatments. These centers provide a controlled environment for administering ERT, ensuring patient safety and comfort during infusions.

Enzyme Replacement Therapy Market Segmentation

Grand View Research has segmented the global enzyme replacement therapy market based on product, therapeutic condition, route of administration, end-use, and region:

Enzyme Replacement Therapy Product Outlook (Revenue, USD Million, 2018 - 2030)

Imiglucerase

Agalsidase Beta

Alglucosidase Alfa

Taliglucerase

Velaglucerase Alfa

Pegademase

Laronidase

Pancreatic Enzymes

Idursulfase

Galsulfase

Others

Enzyme Replacement Therapy Therapeutic Condition Outlook (Revenue, USD Million, 2018 - 2030)

Gaucher Disease

MPS

SCID

Pompe Disease

Fabry Disease

Others

Enzyme Replacement Therapy Route of Administration Condition Outlook (Revenue, USD Million, 2018 - 2030)

Parenteral

Oral

Enzyme Replacement Therapy End-use Outlook (Revenue, USD Million, 2018 - 2030)

Infusion Centers

Hospitals

Others

Enzyme Replacement Therapy Regional Outlook (Revenue, USD Million, 2018 - 2030)

North America

US

Canada

Mexico

Europe

Germany

UK

France

Italy

Spain

Denmark

Sweden

Norway

Asia Pacific

China

India

Japan

Australia

South Korea

Thailand

Latin America

Brazil

Argentina

Middle East & Africa

South Africa

Saudi Arabia

UAE

Kuwait

List of Key Players in Enzyme Replacement Therapy Market

Shire Plc; Sanofi S.A.

Biomarin Pharmaceutical Inc.

AbbVie

Alexion Pharmaceuticals Inc.

Allergan plc

Horizon Pharma Public Limited Company

Actelion (Janssen)

Recordati Rare Diseases

Protalix Biotherapeutics

Amicus Therapeutics, Inc.

Order a free sample PDF of the Enzyme Replacement Therapy Market Intelligence Study, published by Grand View Research.

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Living with Pompe: A Journey Through Diagnosis and Hope

The global Pompe disease market is anticipated to grow steadily over the forecast period from 2022 to 2028. According to the report, the market was valued at approximately USD 730 million in 2022 and is expected to reach nearly USD 950 million by 2028, with a compound annual growth rate (CAGR) of over 4%.

What is Pompe Disease?

Pompe disease, also known as glycogen storage disease type II, is a rare genetic disorder caused by mutations in the gene responsible for producing acid alpha-glucosidase (GAA), an enzyme essential for breaking down glycogen. In individuals with Pompe disease, glycogen accumulates in the cells, particularly in muscle tissue, leading to progressive muscle weakness and respiratory issues. The disease has both infantile and late-onset forms, each with different severity and progression rates.

Get Sample pages of Report: https://www.infiniumglobalresearch.com/reports/sample-request/41141

Market Dynamics and Growth Drivers

Key factors contributing to the growth of the global Pompe disease market include:

Increased Awareness and Diagnosis: As awareness of rare genetic diseases increases, more patients are being diagnosed with Pompe disease. Early detection and intervention are critical for managing disease progression, driving demand for diagnostic and therapeutic solutions.

Advances in Gene Therapy and Enzyme Replacement Therapy (ERT): Significant progress in gene therapy and enzyme replacement therapy is enhancing the efficacy of Pompe disease treatment. ERT, which provides the missing enzyme, is a cornerstone of treatment, while new gene therapies hold potential for long-term benefits by addressing the disease at a genetic level.

Growing Research and Development Investments: Biopharmaceutical companies and research institutions are investing in R&D to develop innovative treatments for Pompe disease. This includes efforts to refine existing therapies, reduce side effects, and improve patient outcomes, ultimately driving market growth.

Expansion of Healthcare Infrastructure: Improving healthcare access, particularly in emerging regions, is facilitating the availability of specialized treatments, including those for rare diseases like Pompe disease. Expanding healthcare infrastructure enables more patients to access diagnostic and treatment services.

Regional Analysis

North America: North America, led by the U.S., is a major market for Pompe disease treatment due to a high prevalence of rare disease screening programs, advanced healthcare infrastructure, and substantial investment in research. Government initiatives to support rare disease patients further bolster the market in this region.

Europe: Europe is also a significant contributor to the Pompe disease market, driven by robust healthcare systems and growing patient support networks. Countries such as Germany, France, and the U.K. play a key role due to their emphasis on rare disease research and patient advocacy programs.

Asia-Pacific: Asia-Pacific is expected to experience the fastest growth in the Pompe disease market. The increasing availability of healthcare services, coupled with rising awareness and improving diagnostic capabilities, particularly in countries like Japan, China, and South Korea, contributes to market expansion.

Latin America, Middle East & Africa: These regions are experiencing gradual growth as awareness and access to specialized healthcare improve. Government and non-government organizations are working to enhance the diagnosis and treatment infrastructure for rare diseases, supporting market growth.

Competitive Landscape

The Pompe disease market is competitive, with a few key players focusing on novel therapeutic development, strategic collaborations, and clinical trials to maintain their market position. Major players include:

Sanofi Genzyme: Sanofi’s Lumizyme (alglucosidase alfa) is one of the most widely used treatments for Pompe disease. Sanofi Genzyme has a strong presence in the market, focusing on expanding access to its enzyme replacement therapies.

Amicus Therapeutics: Known for its product Galafold for Fabry disease, Amicus is actively developing therapies for Pompe disease, including investigational gene therapies, and is working to strengthen its position in the rare disease market.

Audentes Therapeutics: Audentes is exploring gene therapy for Pompe disease and other rare genetic conditions. The company aims to provide long-term solutions to patients by targeting the genetic root of the disease.

Spark Therapeutics: As part of Roche, Spark is advancing gene therapy solutions for a range of genetic disorders, including Pompe disease, with a focus on innovative approaches that provide potentially curative treatments.

Report Overview : https://www.infiniumglobalresearch.com/reports/global-pompe-disease-market

Challenges and Opportunities

The Pompe disease market faces challenges such as high treatment costs, limited awareness in developing regions, and the complexity of gene therapies. Enzyme replacement therapies can be prohibitively expensive, making access difficult for some patients, particularly in regions with limited healthcare funding.

However, the market also presents significant opportunities, including advancements in gene therapy, which offer the potential for more effective, long-term solutions. Research in enzyme replacement and gene therapies continues to uncover new treatment possibilities, fostering optimism for improved patient outcomes.

Conclusion

The global Pompe disease market is set for steady growth, increasing from USD 730 million in 2022 to nearly USD 950 million in 2028 at a CAGR of over 4%. With rising awareness, advancements in treatment options, and increased investment in rare disease research, the Pompe disease market holds promise for improved patient outcomes and a better quality of life for those affected by this rare condition.

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The global market for Pompe disease therapeutics is projected to expand at a compound annual growth rate (CAGR) of 5.00%, from an estimated USD 1584.2 million in 2023 to USD 2457.61 million in 2032.Pompe disease, a rare inherited lysosomal storage disorder, has garnered significant attention within the medical community due to its severe implications and the complexity of its treatment. The disease, characterized by the buildup of glycogen in the body's cells, leads to progressive muscle weakness and respiratory difficulties, often proving fatal without appropriate intervention. The global market for Pompe disease therapeutics has been evolving rapidly, driven by advances in biotechnology, increasing awareness, and growing investments in rare disease research. This article delves into the current trends, challenges, and future prospects of the Pompe disease therapeutics market.

Browse the full report at https://www.credenceresearch.com/report/global-pompe-disease-therapeutics-market

Market Overview

The global Pompe disease therapeutics market is primarily dominated by enzyme replacement therapy (ERT), which has been the cornerstone of treatment since its introduction. Myozyme (alglucosidase alfa), developed by Sanofi Genzyme, remains the leading ERT for Pompe disease, significantly improving patient outcomes, especially in infantile-onset Pompe disease. However, the high cost of ERT, coupled with the lifelong need for treatment, presents substantial economic challenges for patients and healthcare systems.

In addition to ERT, the market is witnessing the emergence of gene therapy as a promising treatment option. Gene therapy aims to correct the underlying genetic defect responsible for Pompe disease by introducing a functional copy of the GAA gene. Several companies are currently in various stages of clinical trials, with AT845 (Astellas Gene Therapies) and ACTUS-101 (Actus Therapeutics) being notable examples. These advancements underscore a shift towards more innovative and potentially curative treatment modalities.

Key Market Drivers

1. Technological Advancements: The rapid progress in genetic engineering and biotechnology has been instrumental in the development of novel therapeutics for Pompe disease. Gene therapy, in particular, is expected to revolutionize the treatment landscape by offering a one-time, potentially curative option.

2. Increasing Awareness and Diagnosis: Growing awareness among healthcare professionals and patients has led to earlier diagnosis and treatment of Pompe disease. Newborn screening programs, particularly in developed countries, have been pivotal in identifying Pompe disease early, allowing for timely intervention.

3. Rising Investment in Rare Disease Research**: The pharmaceutical industry's increasing focus on rare diseases has spurred significant investment in the development of therapies for Pompe disease. This is reflected in the growing number of clinical trials and the entry of new market players, including biotech startups and established pharmaceutical companies.

Challenges Facing the Market

1. High Treatment Costs: The high cost of existing treatments, particularly ERT, remains a significant barrier to access, especially in low- and middle-income countries. The affordability of new therapies, including gene therapy, will be a crucial factor in determining their market penetration.

2. Regulatory Hurdles: The regulatory approval process for novel therapies, particularly gene therapies, is complex and stringent. Ensuring the safety and efficacy of these treatments requires extensive clinical trials, which can delay market entry and increase development costs.

3. Limited Patient Population: As a rare disease, Pompe disease has a limited patient population, which can restrict the commercial viability of new therapeutics. This makes it challenging for companies to recoup the high costs associated with drug development.

Regional Market Insights

The United States and Europe currently dominate the Pompe disease therapeutics market, driven by robust healthcare infrastructure, high awareness, and supportive regulatory frameworks. These regions have been at the forefront of newborn screening programs, which have played a critical role in early diagnosis and treatment initiation. Asia-Pacific, however, is emerging as a key market due to increasing healthcare expenditure, rising awareness, and improving access to advanced therapies.

Future Prospects

The future of the Pompe disease therapeutics market looks promising, with ongoing research and development expected to yield more effective and accessible treatments. Gene therapy holds the potential to transform the treatment paradigm, offering hope for a cure rather than lifelong management. Additionally, advancements in personalized medicine may lead to more tailored and effective treatment approaches.

Key Players

Amicus Therapeutics, Inc. (U.S.)

Audentes Therapeutics (U.S.)

Novartis AG (Switzerland)

Fresenius Kabi AG (Germany)

Akorn Incorporated (U.S.)

Teva Pharmaceutical Industries Ltd (Israel)

Mylan N.V (U.S.)

Johnsons & Johnsons Services Inc (U.S.)

F. Hoffman-La Roche Ltd. (Switzerland)

Danaher (U.S.)

B.D. (U.S.)

Chembio Diagnostics (U.S.)

EKF Diagnostics (U.K.)

Trinity Biotech plc (Ireland)

Instrumentation Laboratory (U.S.)

Nova Biomedical (U.S.)

PTS Diagnostics (U.S.)

Sekisui Diagnostics (U.S.)

Thermo Fisher Scientific (U.S.)

bioMérieux S.A. (France)

Others

Segmentation

By Therapy Type

Enzyme Replacement Therapy (ERT)

Recombinant Enzymes

Long-Acting ERT

Novel ERT Formulations

Gene Therapy

Viral Vector-Based Gene Therapy

Non-Viral Gene Therapy

Small Molecule Therapies

Chaperone Therapies

Substrate Reduction Therapies (SRT)

Supportive Therapies

Symptomatic Treatment

Nutritional Support

By Route of Administration

Intravenous (IV) Administration

Oral Administration

By End-User

Hospitals and Clinics

Specialty Clinics

Home Care Settings

By Region

North America

U.S.

Canada

Mexico

Europe

Germany

France

UK.

Italy

Spain

Rest of Europe

Asia Pacific

China

Japan

India

South Korea

South-east Asia

Rest of Asia Pacific

Latin America

Brazil

Argentina

Rest of Latin America

Middle East & Africa

GCC Countries

South Africa

The Rest of the Middle East and Africa

Browse the full report at https://www.credenceresearch.com/report/global-pompe-disease-therapeutics-market

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Newborn Screening Market Analysis, Market Size, In-Depth Insights, Growth and Forecast 2029

The newborn screening test helps in diagnosing rare diseases in newborns, facilitating early treatment. A steady rise in birth rate coupled with rising incidence of serious health disorders among newborns has amplified the demand for newborn screening around the world. Introduction of favourable government programs & legislations is contributing to the demand for newborn screening. Rise in disposable income and provided affordability is making newborn screening more accessible. These aspects, coupled with cutting-edge developments in the health screening domain have propelled significant growth across the global newbron screening market. Besides, parents have become more conscious of the health of their infants further accelerating the pace of market expansion. All these trends hint at the presence of lucrative business possibilities in the global newborn screening market, over the forecast period.  

 For More Industry Insight Read: https://www.fairfieldmarketresearch.com/report/newborn-screening-market

 An Upswing in Health Disorders to Facilitate Newborn Screening Market Expansion

As people are becoming more informed of the health benefits associated with newborn screening, the demand has also mounted at a significant rate. Some of the most prominent newborn screening tests include tests for metabolic disorders, haemoglobin (red blood cell) conditions, hormone (endocrine) disorders, and immune system disorders. Besides, other rare conditions such as Pompe disease, cystic fibrosis, spinal muscular atrophy and X-linked adrenoleukodystrophy are also being significantly diagnosed. An incessant rise in such serious health conditions among infants is creating substantial demand for newborn screening at a global level. Uptick in demand and prevailing serious health disorders are likely to place newborn screening market on a sound growth trajectory.  

 Growing Incidence of Congenital Heart Abnormalities to Create Significant Growth Prospects for Newborn Screening Market

The rising cases of congenital heart abnormalities including tricuspid atresia and pulmonary atresia globally are driving the newborn screening market. Critical congenital heart defects are the most serious congenital heart defects and newborns with critical CHDs require surgery or other treatment within the first year of their life. Therefore, healthcare experts are advocating and supporting the need for more sophisticated screening tools. Furthermore, medical advancements that include ongoing drug discovery, government and non-government organisational initiatives have promoted awareness regarding congenital heart abnormalities. Considering these factors, the global newborn screening market is set to register fruitful outcomes in revenue generation and business expansion.

 North America to Emerge as Key Region Owing to Favourable Reimbursement Policies

The growing prevalence of congenital disorders and mandatory testing in all states are key factors cementing North America as a key revenue generator in the global newborn screening market. In addition to this, the advancements in healthcare infrastructure are projected to bode well for the market. Other factors boosting the growth of the regional market include favourable reimbursement policies and the available newborn screening programs. The cumulative force of these factors is projected to unlock new revenue opportunities for the newborn screening market in North America.

 Leading Market Players

Some of the global players in the newborn screening market include Trivitron Healthcare (India), PerkinElmer (US), Natus Medical (US), Danaher Corporation (US), Demant A/S (Denmark), Bio-Rad Laboratories (US), Chromsystems Instruments & Chemicals GmbH (Germany), and Baebies (US), Medtronic (Ireland), and Recipe Chemicals+Instruments (Germany).

 For More Information Visit: https://www.fairfieldmarketresearch.com/report/newborn-screening-market

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Fairfield Market Research is a UK-based market research provider. Fairfield offers a wide spectrum of services, ranging from customized reports to consulting solutions. With a strong European footprint, Fairfield operates globally and helps businesses navigate through business cycles, with quick responses and multi-pronged approaches. The company values an eye for insightful take on global matters, ably backed by a team of exceptionally experienced researchers. With a strong repository of syndicated market research reports that are continuously published & updated to ensure the ever-changing needs of customers are met with absolute promptness.

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Global Pompe Disease Treatment Market 2017- Amicus Therapeutics, Audentes Therapeutics, Sanofi Genzyme, EpiVax, Oxyrane

Global Pompe Disease Treatment Market 2017- Amicus Therapeutics, Audentes Therapeutics, Sanofi Genzyme, EpiVax, Oxyrane

Global Pompe Disease Treatment Market Report 2017 offers comprehensive research study on the current scenario of the Pompe Disease Treatment market globally, offering a primary overview of Pompe Disease Treatment market, consisting of definitions, classifications, a range of applications and Pompe Disease Treatment industry chain structure.

Pompe Disease Treatment Market report includes…

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Enzyme Replacement Therapy Market Size USD 13,776.2 million by 2028

According to the latest research published by Future Market Insights, the global enzyme replacement therapy market size is poised to expand at 6.5% CAGR over the forecast period 2022-2028. The global market for enzyme replacement therapy is expected to exceed US$ 13,776.2 Mn by 2028 end, attesting the highest growth rate by Japan, which is expected to expand at 12.5% CAGR over 2022-2028.

Increasing per capita healthcare expenditure in North America is expected to push market revenue growth. The rapidly developing healthcare infrastructure and increasing awareness programmes in the highly populous regions of Asia Pacific and China is expected to play an important role in driving revenue growth of the global enzyme replacement therapy market.

Companies in the global ERT market are focussing on strategic initiatives such as alliances and agreements, collaborations, increasing R&D activities, new product launches, joint ventures, partnerships, and mergers & acquisitions to sustain in the competitive global market.

Key Research Findings

Enzyme replacement therapy is used in the treatment of rare diseases and the global market is projected to create incremental opportunity of US$ 6,448.3 Mn between 2022 and 2028

When compared to other established markets such as North America, the enzyme replacement therapy markets in Japan and Western Europe are expected to witness fast and steady growth, partly due to the increasing governmental support and growing demand for enzyme replacement therapy for the treatment of rare diseases.

Global Enzyme Replacement Therapy Market: Competitive Landscape

The global market is highly consolidated owing to the presence of several players. Some of the key players are Sanofi (Genzyme Corporation), Shire, Pfizer, Inc., Alexion Pharmaceuticals, Inc., BioMarin Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc., Johnson & Johnson Services, Inc., Allergan, Leadiant Biosciences, Inc., and others.

Key Segments of Enzyme Replacement Therapy Industry Survey

Enzyme Replacement Therapy Market by Therapeutic Condition:

Enzyme Replacement Therapy for Fabry Disease

Enzyme Replacement Therapy for Gaucher Disease

Enzyme Replacement Therapy for Mucopolysaccharidosis

Enzyme Replacement Therapy for Pompe Disease

Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency

Others

Enzyme Replacement Therapy Market by Route of Administration:

Oral Enzyme Replacement Therapy

Injectable Enzyme Replacement Therapy

Enzyme Replacement Therapy Market by Distribution Channel:

Hospital Pharmacies

Specialty Treatment Pharmacies

Retail Pharmacies

Enzyme Replacement Therapy Market by Region:

North America Enzyme Replacement Therapy Market

Latin America Enzyme Replacement Therapy Market

Europe Enzyme Replacement Therapy Market

East Asia Enzyme Replacement Therapy Market

South Asia & Pacific Enzyme Replacement Therapy Market

Middle East & Africa (MEA) Enzyme Replacement Therapy Market

Information Source: https://www.futuremarketinsights.com/reports/enzyme-replacement-therapy-market

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The “Pompe Disease Treatment  Market ”report provides a detailed analysis of global market size, regional and country-level market size, segmentation, market growth, market share, and competitive Landscape.

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“Pompe Disease Treatment Market ”gives detailed outlook by Type, by Application, by Segmentation and Regional Forecasts.

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Pompe Disease Treatment Market Size Industry Insights, Top Trends, Drivers, Growth and Forecast to 2027

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Enzyme Replacement Therapy Market Size is expected to exceed US$ 13,776.2 million by 2028 | FMI Research

According to the latest research published by Future Market Insights, the Enzyme Replacement Therapy Market Size is poised to expand at 6.5% CAGR over the forecast period 2022-2028. The global market for enzyme replacement therapy is expected to exceed US$ 13,776.2 Mn by 2028 end, attesting the highest growth rate by Japan, which is expected to expand at 12.5% CAGR over 2022-2028.

Increasing per capita healthcare expenditure in North America is expected to push market revenue growth. The rapidly developing healthcare infrastructure and increasing awareness programmes in the highly populous regions of Asia Pacific and China is expected to play an important role in driving revenue growth of the global enzyme replacement therapy market.

Companies in the global ERT market are focussing on strategic initiatives such as alliances and agreements, collaborations, increasing R&D activities, new product launches, joint ventures, partnerships, and mergers & acquisitions to sustain in the competitive global market.

Key Research Findings

Enzyme replacement therapy is used in the treatment of rare diseases and the global market is projected to create incremental opportunity of US$ 6,448.3 Mn between 2022 and 2028

When compared to other established markets such as North America, the enzyme replacement therapy markets in Japan and Western Europe are expected to witness fast and steady growth, partly due to the increasing governmental support and growing demand for enzyme replacement therapy for the treatment of rare diseases.

Global Enzyme Replacement Therapy Market: Competitive Landscape

The global market is highly consolidated owing to the presence of several players. Some of the key players are Sanofi (Genzyme Corporation), Shire, Pfizer, Inc., Alexion Pharmaceuticals, Inc., BioMarin Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc., Johnson & Johnson Services, Inc., Allergan, Leadiant Biosciences, Inc., and others.

Key Segments of Enzyme Replacement Therapy Industry Survey

Enzyme Replacement Therapy Market by Therapeutic Condition:

Enzyme Replacement Therapy for Fabry Disease

Enzyme Replacement Therapy for Gaucher Disease

Enzyme Replacement Therapy for Mucopolysaccharidosis

Enzyme Replacement Therapy for Pompe Disease

Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency

Others

Enzyme Replacement Therapy Market by Route of Administration:

Oral Enzyme Replacement Therapy

Injectable Enzyme Replacement Therapy

Enzyme Replacement Therapy Market by Distribution Channel:

Hospital Pharmacies

Specialty Treatment Pharmacies

Retail Pharmacies

Enzyme Replacement Therapy Market by Region:

North America Enzyme Replacement Therapy Market

Latin America Enzyme Replacement Therapy Market

Europe Enzyme Replacement Therapy Market

East Asia Enzyme Replacement Therapy Market

South Asia & Pacific Enzyme Replacement Therapy Market

Middle East & Africa (MEA) Enzyme Replacement Therapy Market

Information Source: https://www.futuremarketinsights.com/reports/enzyme-replacement-therapy-market

[marketnewsreport]

Pompe Disease Treatment Market is Booming Market to Grow at Healthy CAGR forecast by 2028

pompe disease treatment market is expected to gain market growth in the forecast period of 2021-2028.Pompe disease is a genetic disorder that is caused by the buildup of a complex sugar called glycogen in cells and tissues. This accumulation of glycogen causes impairment in organs and tissues.

This disease majorly causes muscle wasting, muscle weakness and other complications such as cardiac problems and respiratory disorders. GAA gene is responsible for breakdown of glycogen in the body. In Pompe disease, mutation occurs in GAA gene that results in accumulation of glycogen in tissues and organs.

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Key Market Players:

Few of the major competitors currently working in the global pompe disease treatment market are Amicus Therapeutics, Inc., Takeda Pharmaceutical Company, Sanofi,, Valerion Therapeutics, Sangamo Therapeutics, Oxyrane, EpiVax, Inc., Alexion Pharmaceuticals, Inc., Novartis AG, GlaxoSmithKline plc., Audentes Therapeutics, Spark Therapeutics, Inc., Pfizer Inc., BioMarin, ABIOMED, Sarepta Therapeutics,. Biogen, Genentech, Inc., among others.

Segmentation: Global Pompe Disease Treatment Market

·         By Type (Classic Infantile-Onset Pompe Disease, Non-Classic Infantile-Onset Pompe Disease, Late-Onset Pompe Disease)

·         By Therapy Type (Medication, Enzyme Replacement Therapy, Physiotherapy, Others)

·         By Route of Administration (Oral, Injectable, Others)

·         By End Users (Hospitals, Homecare, Specialty Clinics, Others)

·         By Distribution Channel (Hospital Pharmacy, Online Pharmacy, Retail Pharmacy, Others)

·         By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa)

Competitive Analysis:

Global pompe disease treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of Pompe disease treatment market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Market Drivers

·         Advancement in the gene therapy and enzyme replacement therapies also acts as a market driver

·         Rising healthcare infrastructure for Pompe disease drugs will expand the market size

·         Increasing research and development for effective treatments and diagnosis technologies for Pompe disease is another factor boosting the market growth

·         Rising prevalence rate of people suffering from Pompe disease is expected to drive the market growth in the forecast period

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Market Restraints

·         Limited patient pool and heterogeneity of the disease in different regions hampers the market growth

·         Lack of awareness about the disease and its available treatment options are major restrains for the market growth

·         High cost associated with the therapy will also restrict the market growth

Reasons to Purchase this Report

·         Current and future of global pompe disease treatment market outlook in the developed and emerging markets

·         The segment that is expected to dominate the market as well as the segment which holds highest CAGR in the forecast period

·         Regions/Countries that are expected to witness the fastest growth rates during the forecast period

·         The latest developments, market shares, and strategies that are employed by the major market players

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