ムコ多糖症治療市場レポート：動向、予測、競合分析 (2031年まで)

世界のムコ多糖症治療市場の将来は、病院と専門クリニック市場での機会で有望視されています。世界のムコ多糖症治療市場は、2025年から2031年までのCAGRが8.4%で、2031年までに推定46億米ドルに達すると予想されています。この市場の主な促進要因は、効果的な治療に対する需要の増加、ムコ多糖症の有病率の上昇、医療支出の増加です。

• Lucintel社の予測によると、治療法別では酵素補充療法が、MPS患者の症状やQOLの改善に役立ち、寿命も延ばせることから、病院や診療所からの需要が高く、引き続き大きなセグメントとなります。
• 最終用途別の中では病院が、MPS治療を提供するために必要な専門的な検査室や設備などのインフラや、訓練を受けた医療専門家などの専門知識を有しているため、予測期間中も最大セグメントであり続けると思われます。
• 地域別では、確立された医療制度とMPS償還政策に支えられたMPSの高い有病率により、北米が最大のセグメントであり続けると思われます。

ムコ多糖症治療市場の戦略的成長機会

ムコ多糖症治療市場は、既存の治療法の新たな応用の模索や新規治療アプローチの開発により、多くの成長見込みがあります。ムコ多糖症という疾患に対する認識は、遺伝子治療、酵素補充療法、個別化医療など、多様な用途における主要な成長機会の特定を後押しし、異なるMPSサブタイプにおける特定のニーズのターゲティングを強化しています。

• 遺伝子治療向け用途の拡大：従来の酵素補充療法にとどまらない遺伝子治療向け用途の拡大は、MPS市場に大きな成長機会をもたらしています。現在進行中の調査と臨床試験により、遺伝子治療はMPS治療の長期的ソリューションとして有望視されています。この革新的なアプローチは、疾患の根底にある遺伝的欠陥を修正し、患者の転帰を改善しながら生涯にわたる介入の必要性を排除する可能性を秘めています。この成長機会は多額の投資を呼び込み、多様なMPSサブタイプをターゲットとする新たな遺伝子治療の開発につながると期待されています。
• 新しい酵素補充療法の開発：MPS治療市場は、主要な成長分野として、より効率的で副作用の少ない新規ERTの創出から利益を得ることができます。企業は、この疾患によく罹患する脳など、特定の組織や臓器により特異的に作用する次世代ERTの開発に関心を寄せています。ERTのこれらの開発により、患者のQOLが向上し、これらの薬剤の使用が拡大し、より多くの人々がより手頃な価格で使用できるようになると予想されます。
• 新生児スクリーニングプログラムの拡大：MPSを含む新生児スクリーニングプログラムの拡大は、早期発見と早期介入を可能にするもう一つの成長機会です。このようなプログラムが他国でも広く実施されることで、早期段階での診断機器や治療薬に対する需要が高まる可能性があります。このような成長の可能性は、早期治療を可能にすることで患者のケアを向上させ、疾患の早期段階での診断や治療が増えることで市場を拡大させます。
• 研究開発投資の増加：MPS治療に関する研究開発（R&D）への投資の増加は、重要な成長機会です。製薬会社や研究機関が新しい治療法の開発や既存の治療法の改良にさらに力を入れるようになれば、この分野には革新的な治療法が次々と生み出されると思われます。このような投資により、新たな治療戦略が見出され、患者が利用できる治療の選択肢が増え、ひいては市場の成長につながると予想されます。

遺伝子治療の進歩、個別化医療、幅広いスクリーニングプログラム、研究開発投資の増加、共同研究は、ムコ多糖症治療市場の戦略的成長機会に影響を与える主な要因です。これらの選択肢は利用可能な治療法の種類を増やし、市場の大幅な拡大を促進しています。このような展望が実現しつつあることで、希少疾患治療の展望の中で重要な重点分野の一つとして患者に新たな生命をもたらし、ムコ多糖症治療市場の成長につながると期待されています。

ムコ多糖症治療市場の促進要因・課題

ムコ多糖症治療市場は、その成長と開拓に影響を与えるいくつかの市場促進要因・課題によって形成されています。主な促進要因としては、医療技術の進歩、希少疾患に対する認知度の向上、医療インフラの拡大などが挙げられます。一方、重大なハードルとしては、高額な治療費、発展途上国における治療法へのアクセスの制限、希少疾患や遺伝的に不均一な疾患の治療に伴う複雑さなどが挙げられます。

ムコ多糖症治療市場を牽引する要因は以下の通りです：

• 医療技術の進歩：遺伝子治療や次世代酵素補充療法など、より効率的な治療法の開発が可能になりました。こうした技術革新は患者の予後を改善し、治療の選択肢を広げるため、新たな治療法の登場とともに市場の成長につながります。
• 希少疾患に対する意識の高まり：MPSを含む希少疾患に対する認識の高まりは、診断法や治療法に対する需要の増加に寄与しています。擁護活動や教育プログラムが早期発見や治療の改善につながり、患者基盤の拡大とそれに伴う市場規模の拡大につながっています。
• 医療インフラの拡大：特に発展途上地域における医療・インフラの開発により、より多くの人々がMPS治療にアクセスできるようになりました。医療水準の向上により、より多くの患者がMPSの専門医療を受けられるようになるため、治療ソリューションや検査ツールに対する需要は今後も増加し続けると思われます。

ムコ多糖症治療市場における課題は以下の通りです：

• 高い治療費：遺伝子治療や酵素補充療法を含むMPS治療は高額であり、患者や医療システムにとって大きな課題となります。その結果、特に新興諸国では、コストの問題からこれらの治療法へのアクセスが制限される可能性があり、市場開拓の障壁となっています。
• 開発途上地域における治療法へのアクセス制限：不十分な医療インフラ、高額な費用、認知度の低さにより、発展途上地域ではMPS治療へのアクセスが限られています。この問題は市場拡大の妨げとなり、十分な医療が行き届いていない地域全体で重要な薬剤へのアクセスを向上させる方法の必要性を浮き彫りにしています。
• 希少かつ多様な疾患の治療の複雑さ：MPSは遺伝的に多様な疾患群から構成されており、効果的な治療法を開発・提供する上での課題となっています。様々な病態があるため専門的な治療が必要となり、大規模なニーズへの対応が困難で、市場拡大の妨げとなっています。

ムコ多糖症治療市場は、技術の進歩、知識の増加、強固な医療システム、研究開発費の増加、支援的規制によって牽引されてきました。しかし、治療費の高騰、低開発国でのアクセス制限、希少かつ異種疾患であるムコ多糖症の管理の複雑さといった課題にも直面しています。こうした市場促進要因・課題が市場の軌跡を形成し、世界中の患者にとって利用しやすく有効であることを保証するために必要な新治療法や戦略の開発に影響を与えています。

目次

第1章 エグゼクティブサマリー

第2章 世界のムコ多糖症治療市場：市場力学

• イントロダクション、背景、分類
• サプライチェーン
• 業界の促進要因と課題

第3章 市場動向と予測分析 (2019年～2031年)

• マクロ経済動向 (2019～2024年) と予測 (2025～2031年)
• 世界のムコ多糖症治療市場の動向 (2019～2024年) と予測 (2025～2031年)
• 世界のムコ多糖症治療市場：種類別
  • MPS-I
  • MPS-II
  • MPS-IV
  • MPS-VI
  • MPS-VII
  • その他
• 世界のムコ多糖症治療市場：治療法別
  • 酵素補充療法
  • 幹細胞療法
• 世界のムコ多糖症治療市場：最終用途別
  • 病院
  • 専門クリニック
  • その他

第4章 地域別の市場動向と予測分析 (2019年～2031年)

• 世界のムコ多糖症治療市場：地域別
• 北米のムコ多糖症治療市場
• 欧州のムコ多糖症治療市場
• アジア太平洋のムコ多糖症治療市場
• その他地域のムコ多糖症治療市場

第5章 競合分析

• 製品ポートフォリオ分析
• 運用統合
• ポーターのファイブフォース分析

第6章 成長機会と戦略分析

• 成長機会分析
  • 世界のムコ多糖症治療市場の成長機会：種類別
  • 世界のムコ多糖症治療市場の成長機会：治療法別
  • 世界のムコ多糖症治療市場の成長機会：最終用途別
  • 世界のムコ多糖症治療市場の成長機会：地域別
• 世界のムコ多糖症治療市場の新たな動向
• 戦略的分析
  • 新製品の開発
  • 世界のムコ多糖症治療市場の生産能力拡大
  • 世界のムコ多糖症治療市場における企業合併・買収 (M&A)、合弁事業
  • 認証とライセンシング

第7章 主要企業のプロファイル

• Take da Pharmaceutical
• BioMarin Pharmaceuticals
• Ultragenyx Pharmaceutical
• Sarepta Therapeutics
• Abeona Therapeutics
• Eloxx Pharmaceuticals
• Esteve
• Immusoft Corporation
• Inventiva
• GC Pharma

The future of the global mucopolysaccharidosis treatment market looks promising with opportunities in the hospital and specialty clinic markets. The global mucopolysaccharidosis treatment market is expected to reach an estimated $4.6 billion by 2031 with a CAGR of 8.4% from 2025 to 2031. The major drivers for this market are the increasing demand for effective treatments, the rising prevalence of mucopolysaccharidosis, and rising healthcare spending.

• Lucintel forecasts that, within the treatment category, enzyme replacement therapy will remain the larger segment due to high demand from hospitals and clinics as it helps to improve the symptoms and quality of life for MPS patients, and it can also extend their lifespan.
• Within the end use category, hospitals will remain the largest segment over the forecast period because it has the necessary infrastructure, such as specialized laboratories and equipment, and the necessary expertise like trained healthcare professionals, to provide MPS treatment.
• In terms of regions, North America will remain the largest segment due to the high prevalence of MPS supported by a well-established healthcare system and MPS reimbursement policy.

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Emerging Trends in the Mucopolysaccharidosis Treatment Market

The mucopolysaccharidosis treatment market is undergoing significant changes driven by emerging trends that define the future of care for patients with these rare genetic disorders. These trends have been influenced by progress in biotechnology, rising global awareness, and a changing regulatory environment. Understanding these trends is crucial for stakeholders in the MPS treatment market as they shed light on areas of research focus and development, which may result in improved or new drugs that can better suit patients' needs.

• Increase in Gene Therapy Research: Gene therapy is emerging as a potential trend in the MPS treatment industry, with the possibility of addressing diseases at their root for long-term solutions. This area of research has witnessed remarkable growth, currently involving several clinical trials aimed at determining the suitability and safety of gene therapies for different types of MPS. These interventions help correct defects traced back to changes in genes by introducing functional ones, potentially offering a single treatment route with a high chance of better patient outcomes. The results obtained from these tests could be miraculous, serving as signs of hope for finding a cure for MPS.
• Progress in Enzyme Replacement Therapies (ERTs): Enzyme replacement therapies (ERTs) have remained the pillar of MPS treatment, augmented through continuous improvement initiatives. Patient experience can be enhanced through new formulations and methods of drug administration, leading to reduced administration rates and increased efficacy for ERTs. Due to these improvements, it is becoming easier to use enzyme replacement treatments not only in hospital settings but also at home, making ERTs particularly important for the long-term care of patients suffering from MPS.
• Expanding Newborn Screening Programs for MPS: The expansion of newborn screening programs for MPS is a crucial trend that enhances early diagnosis and intervention. Early detection allows for the timely initiation of treatment, which can greatly improve the prognosis for patients with MPS. Supported by developments in diagnostic technologies, these screening programs are increasingly becoming prevalent worldwide. This wave is expected to lead to better patient outcomes and reduced disease burden, as early interventions can prevent or mitigate many severe complications related to MPS.
• Increasing Global Awareness and Advocacy: There is a growing global awareness and advocacy for MPS through patient organizations, healthcare professionals, and social media, boosting their visibility. These efforts help the public understand more about MPS while informing policymakers on how to improve access to treatments and support research funding. Through advocacy initiatives, comprehensive care models have been developed to improve the quality of life for individuals living with MPS. As this awareness continues to grow, it is projected to drive further progress in the management and treatment of MPS.

The unfolding trends in the MPS treatment market, ranging from gene therapy research to personalized medicine, are changing how patients with these rare genetic disorders are cared for. These changes will continue evolving into greater improvements in treatment options, making therapies better, more accessible, and individually focused to meet patients' needs. There are prospects for better treatments for MPS due to ongoing research and innovation that could lead to discoveries along incredible paths.

Recent Developments in the Mucopolysaccharidosis Treatment Market

The mucopolysaccharidosis treatment market is experiencing significant advancements as new therapies and approaches are developed to manage and treat this group of rare, inherited metabolic disorders. With increased investment in research, advancements in gene therapy, and greater awareness among healthcare professionals, the landscape for mucopolysaccharidosis (MPS) treatment is rapidly evolving. These developments are not only expanding the treatment options available to patients but are also paving the way for more personalized and effective therapies.

• Advancements in Gene Therapy: Gene therapy is emerging as a promising treatment for MPS, offering the potential to address the underlying genetic defects responsible for the disorder. Recent clinical trials have shown positive results, with some therapies moving into advanced stages of development. These advancements are significant as they provide a potential one-time treatment option that could greatly improve patient outcomes. The impact of gene therapy on the MPS market is expected to be transformative, potentially reducing the need for lifelong enzyme replacement therapies and other ongoing treatments.
• Improved Enzyme Replacement Therapies (ERT): The replacement of enzymes via infusion is the most significant treatment approach for MPS patients, but this is now being improved to enhance the efficacy and versatility of enzyme replacement therapies. New formulations are being developed that will improve bioavailability as well as reduce the frequency of administration. Moreover, research is being conducted to address certain limitations related to ERT, such as its inability to cross the blood-brain barrier. These changes in ERT predict significant improvements in compliance and outcomes, thus cementing its place as a management strategy for MPS.
• Increased Attention on Early Diagnosis and Screening: Significant improvements have been made in relation to early diagnosis and screening for MPS, including an increase in the global adoption of newborn screening programs by many countries. Advanced diagnostic methods now allow for early detection before any permanent damage occurs. These developments are life-changing, as earlier diagnosis could lead to better disease control, increased quality of life among patients, and reduced healthcare costs over time.
• The Rise of Customized Medication: Personalized medicine, which is increasingly crucial in the treatment of MPS, customizes therapy to fit specific genetic mutations in patients. This enables a more targeted and effective therapeutic approach, reducing side effects and improving patient outcomes. This shift may revolutionize the MPS market regarding personalized treatment protocols, leading to hopes of better disease management and improved long-term results for patients.

The mucopolysaccharidosis treatment market is evolving quickly due to recent developments that will allow for more efficient treatments. Advances in gene therapy, improvements in enzyme replacement therapies, early detection methods, personalized medicine, and collaborative research are rapidly changing the landscape. These developments not only improve the lives of patients but also provide new market opportunities for pharmaceutical companies. Over time, the MPS treatment market may grow significantly and change dramatically as innovative treatments reach more people.

Strategic Growth Opportunities for Mucopolysaccharidosis Treatment Market

The mucopolysaccharidosis treatment market has many growth prospects due to the exploration of new applications of existing therapies and the development of novel treatment approaches. Awareness about the disease enhances targeting of specific needs in different MPS subtypes, boosting the identification of key growth opportunities across diverse applications such as gene therapy, enzyme replacement therapy, or personalized medicine, among others.

• Gene Therapy Application Expansion: The expansion of gene therapy applications beyond traditional enzyme replacement therapies presents a major growth opportunity in the MPS market. Ongoing research and clinical trials make gene therapy promising as a long-term solution for treating MPS. This transformative approach has the potential to correct genetic defects underlying the ailment, obviating the need for lifelong interventions while improving outcomes for patients. This growth opportunity is expected to attract significant investment and lead to the development of new gene therapies targeting diverse MPS subtypes.
• New Enzyme Replacement Therapies Development: The MPS treatment market can benefit from the creation of novel ERTs that are more efficient and have fewer side effects as a key growth area. Companies are interested in developing next-generation ERTs that can be more specific for certain tissues or organs, such as the brain, which is commonly affected by this illness. These developments in ERT are anticipated to enhance patients' quality of life and expand the use of these drugs, making them more affordable for a wider range of people.
• Expansion of Newborn Screening Programs: Expanded newborn screening programs to include MPS represent another opportunity for growth, enabling early identification and intervention. Wider implementation of these programs by other countries may result in higher demand for diagnostic devices and therapies at early stages. This growth possibility improves patient care by allowing for early treatment and extends the market, as there would be more diagnoses and treatments made at earlier stages of the disease.
• Increasing Investment in Research and Development: Increased investment in research and development (R&D) for MPS treatments is a vital growth opportunity. When pharmaceutical firms and research institutions focus more on developing new therapies and improving existing ones, this sector will experience an influx of innovative treatments. This investment is anticipated to result in the identification of fresh therapeutic strategies, increasing the treatment options available for patients, which, in turn, will lead to market growth.

Gene therapy advances, individualized medicine, wider screening programs, increased investments in R&D, and collaboration are the main factors influencing strategic growth opportunities in the mucopolysaccharidosis treatment market. These options are broadening the variety of remedies available and driving significant market expansion. The ongoing realization of these prospects is expected to lead to growth in the MPS treatment market, bringing new life to patients as one of the key focus areas within the landscape for rare disease treatment.

Mucopolysaccharidosis Treatment Market Driver and Challenges

The mucopolysaccharidosis treatment market is shaped by several drivers and challenges that affect its growth and development. Key influencers include advances in medical technology, greater awareness of rare diseases, and an expanding healthcare infrastructure. On the other hand, significant hurdles include high treatment costs, limited access to therapies in developing countries, and the complexities involved in treating rare and genetically heterogeneous diseases.

The factors driving the mucopolysaccharidosis treatment market include:

• Technological Advancements in Medical Technology: Technological advancements are the primary driving factor for the MPS treatment market, enabling the development of more efficient therapies such as gene therapy and next-generation enzyme replacement therapies. These innovations improve patient outcomes and expand the range of therapeutic options, leading to market growth as new treatments become available.
• Heightened Awareness of Rare Diseases: Increasing awareness of rare diseases, including MPS, is contributing to a rise in demand for diagnostic methods and therapies. Advocacy efforts and educational programs have led to earlier detection and improved care, thereby expanding the patient base and boosting market size accordingly.
• Expanding Healthcare Infrastructure: The growth of healthcare infrastructure, especially in developing areas, has made it possible for more people to access MPS treatments. Demand for therapeutic solutions and testing tools will continue to rise as more patients can access specialized MPS medical attention due to improving healthcare standards.

Challenges in the mucopolysaccharidosis treatment market include:

• High Cost of Treatment: MPS treatment, including gene therapy and enzyme replacement therapy, is expensive, posing a significant challenge for patients and healthcare systems. Consequently, access to these therapies may be limited due to cost implications, especially in developing countries, creating barriers to market growth.
• Limited Access to Therapies in Developing Regions: Inadequate healthcare infrastructure, high costs, and low awareness levels contribute to limited access to MPS treatments in developing regions. This problem hampers market expansion and highlights the need for methods to enhance accessibility to these critical drugs across underserved areas.
• Complexities of Treating a Rare and Diverse Disease: MPS comprises a genetically diverse group of diseases that present challenges in developing and delivering effective therapies. The varied presentations require specialized treatments, making it difficult to address needs on a large scale, hindering market expansion.

The mucopolysaccharidosis treatment market has been driven by technological advancements, increased knowledge, robust healthcare systems, rising expenditures in research and development, and supportive regulations. However, it also faces challenges such as high treatment costs, limited accessibility in underdeveloped countries, and the complexities of managing a rare and heterogeneous disorder. These drivers and challenges are shaping the market's trajectory, influencing the development of new therapies and strategies needed to ensure their accessibility and effectiveness for patients worldwide.

List of Mucopolysaccharidosis Treatment Companies

Companies in the market compete on the basis of product quality offered. Major players in this market focus on expanding their manufacturing facilities, R&D investments, infrastructural development, and leverage integration opportunities across the value chain. With these strategies mucopolysaccharidosis treatment companies cater increasing demand, ensure competitive effectiveness, develop innovative products & technologies, reduce production costs, and expand their customer base. Some of the mucopolysaccharidosis treatment companies profiled in this report include-

• Take da Pharmaceutical
• BioMarin Pharmaceuticals
• Ultragenyx Pharmaceutical
• Sarepta Therapeutics
• Abeona Therapeutics
• Eloxx Pharmaceuticals
• Esteve
• Immusoft Corporation
• Inventiva
• GC Pharma

Mucopolysaccharidosis Treatment by Segment

The study includes a forecast for the global mucopolysaccharidosis treatment market by type, treatment, end use, and region.

Mucopolysaccharidosis Treatment Market by Type [Analysis by Value from 2019 to 2031]:

• MPS-I
• MPS-II
• MPS-IV
• MPS-VI
• MPS-VII
• Others

Mucopolysaccharidosis Treatment Market by Treatment [Analysis by Value from 2019 to 2031]:

• Enzyme Replacement Therapy
• Stem Cell Therapy

Mucopolysaccharidosis Treatment Market by End Use [Analysis by Value from 2019 to 2031]:

• Hospitals
• Specialty Clinics
• Others

Mucopolysaccharidosis Treatment Market by Region [Analysis by Value from 2019 to 2031]:

• North America
• Europe
• Asia Pacific
• The Rest of the World

Country Wise Outlook for the Mucopolysaccharidosis Treatment Market

The mucopolysaccharidosis (MPS) treatment market has seen significant advancements in recent years, driven by growing research efforts and increased awareness of these rare genetic disorders. MPS disorders are characterized by the body's inability to break down certain complex carbohydrates, necessitating targeted treatments that are evolving with new therapies and improved diagnostics. This is a global market, with key developments being reported in major regions such as China, Germany, Japan, India, and the United States.

• United States: The United States has made great strides in the treatment of MPS, evidenced by ongoing clinical trials and FDA approvals for novel enzyme replacement therapies (ERTs) and gene therapy. Additionally, there has been an increasing trend toward collaboration between pharmaceutical companies and research institutes to develop groundbreaking treatments. Recent developments include the scaling up of newborn screening programs aimed at the early detection of MPS, enabling timely intervention. Consequently, these activities are helping patients recover faster while promoting the development of personalized therapeutic options tailored to different types of MPS.
• China: MPS is gaining more attention in China as the government invests in research and development to serve patients with such conditions. Programs offering specialized healthcare have been initiated to enhance the diagnosis and management of MPS while increasing the availability of ERTs. Moreover, Chinese biopharmaceutical companies are beginning to explore gene therapy options aimed at delivering improved and long-lasting therapies. These developments have gradually enhanced access to care for MPS patients in China.
• Germany: Germany leads in rare disease research, particularly concerning MPS cases. The country is spearheading advanced therapy development centered on ERT and gene therapy. Additionally, specialized treatment centers for MPS are being established, providing comprehensive care from diagnosis through long-term management. German researchers are also participating in international collaborations to find novel therapeutic avenues and enhance the quality of life for MPS patients. As such, Germany has emerged as a major contributor to the global MPS treatment industry.
• India: Diagnosis of MPS in India has improved significantly, though challenges with treatment access remain. Numerous initiatives by healthcare providers aim to enhance diagnostic services and ERT options in the country. Indian pharmaceutical firms, collaborating with global companies, are facilitating the availability of new therapeutic options in the market. However, accessibility remains a significant issue, especially regarding making treatments more affordable for all potentially affected individuals. Nonetheless, India is progressing in the welfare of MPS patients.
• Japan: Japan has recorded significant milestones in MPS treatment recently, focusing on rare diseases and orphan drugs. Supportive regulatory frameworks facilitating the development and approval of MPS treatment options have been implemented in the country. Japan is also exploring revolutionary treatment strategies such as gene therapy and regenerative medicine, which could be effective for individuals living with MPS. Developments like these include introducing new ERTs and expanding clinical trials for emerging therapies. These activities have improved care standards for Japanese patients with MPS and contributed to global research initiatives.

Features of the Global Mucopolysaccharidosis Treatment Market

Market Size Estimates: Mucopolysaccharidosis treatment market size estimation in terms of value ($B).

Trend and Forecast Analysis: Market trends (2019 to 2024) and forecast (2025 to 2031) by various segments and regions.

Segmentation Analysis: Mucopolysaccharidosis treatment market size by type, treatment, end use, and region in terms of value ($B).

Regional Analysis: Mucopolysaccharidosis treatment market breakdown by North America, Europe, Asia Pacific, and Rest of the World.

Growth Opportunities: Analysis of growth opportunities in different types, treatments, end uses, and regions for the mucopolysaccharidosis treatment market.

Strategic Analysis: This includes M&A, new product development, and competitive landscape of the mucopolysaccharidosis treatment market.

Analysis of competitive intensity of the industry based on Porter's Five Forces model.

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This report answers following 11 key questions:

• Q.1. What are some of the most promising, high-growth opportunities for the mucopolysaccharidosis treatment market by type (MPS-I, MPS-II, MPS-IV, MPS-VI, MPS-VII, and others), treatment (enzyme replacement therapy and stem cell therapy), end use (hospitals, specialty clinics, and others), and region (North America, Europe, Asia Pacific, and the Rest of the World)?
• Q.2. Which segments will grow at a faster pace and why?
• Q.3. Which region will grow at a faster pace and why?
• Q.4. What are the key factors affecting market dynamics? What are the key challenges and business risks in this market?
• Q.5. What are the business risks and competitive threats in this market?
• Q.6. What are the emerging trends in this market and the reasons behind them?
• Q.7. What are some of the changing demands of customers in the market?
• Q.8. What are the new developments in the market? Which companies are leading these developments?
• Q.9. Who are the major players in this market? What strategic initiatives are key players pursuing for business growth?
• Q.10. What are some of the competing products in this market and how big of a threat do they pose for loss of market share by material or product substitution?
• Q.11. What M&A activity has occurred in the last 5 years and what has its impact been on the industry?

Table of Contents

1. Executive Summary

2. Global Mucopolysaccharidosis Treatment Market : Market Dynamics

• 2.1: Introduction, Background, and Classifications
• 2.2: Supply Chain
• 2.3: Industry Drivers and Challenges

3. Market Trends and Forecast Analysis from 2019 to 2031

• 3.1. Macroeconomic Trends (2019-2024) and Forecast (2025-2031)
• 3.2. Global Mucopolysaccharidosis Treatment Market Trends (2019-2024) and Forecast (2025-2031)
• 3.3: Global Mucopolysaccharidosis Treatment Market by Type
  • 3.3.1: MPS-I
  • 3.3.2: MPS-II
  • 3.3.3: MPS-IV
  • 3.3.4: MPS-VI
  • 3.3.5: MPS-VII
  • 3.3.6: Others
• 3.4: Global Mucopolysaccharidosis Treatment Market by Treatment
  • 3.4.1: Enzyme Replacement Therapy
  • 3.4.2: Stem Cell Therapy
• 3.5: Global Mucopolysaccharidosis Treatment Market by End Use
  • 3.5.1: Hospitals
  • 3.5.2: Specialty Clinics
  • 3.5.3: Others

4. Market Trends and Forecast Analysis by Region from 2019 to 2031

• 4.1: Global Mucopolysaccharidosis Treatment Market by Region
• 4.2: North American Mucopolysaccharidosis Treatment Market
  • 4.2.1: North American Mucopolysaccharidosis Treatment Market by Treatment: Enzyme Replacement Therapy and Stem Cell Therapy
  • 4.2.2: North American Mucopolysaccharidosis Treatment Market by End Use: Hospitals, Specialty Clinics, and Others
• 4.3: European Mucopolysaccharidosis Treatment Market
  • 4.3.1: European Mucopolysaccharidosis Treatment Market by Treatment: Enzyme Replacement Therapy and Stem Cell Therapy
  • 4.3.2: European Mucopolysaccharidosis Treatment Market by End Use: Hospitals, Specialty Clinics, and Others
• 4.4: APAC Mucopolysaccharidosis Treatment Market
  • 4.4.1: APAC Mucopolysaccharidosis Treatment Market by Treatment: Enzyme Replacement Therapy and Stem Cell Therapy
  • 4.4.2: APAC Mucopolysaccharidosis Treatment Market by End Use: Hospitals, Specialty Clinics, and Others
• 4.5: ROW Mucopolysaccharidosis Treatment Market
  • 4.5.1: ROW Mucopolysaccharidosis Treatment Market by Treatment: Enzyme Replacement Therapy and Stem Cell Therapy
  • 4.5.2: ROW Mucopolysaccharidosis Treatment Market by End Use: Hospitals, Specialty Clinics, and Others

5. Competitor Analysis

• 5.1: Product Portfolio Analysis
• 5.2: Operational Integration
• 5.3: Porter's Five Forces Analysis

6. Growth Opportunities and Strategic Analysis

• 6.1: Growth Opportunity Analysis
  • 6.1.1: Growth Opportunities for the Global Mucopolysaccharidosis Treatment Market by Type
  • 6.1.2: Growth Opportunities for the Global Mucopolysaccharidosis Treatment Market by Treatment
  • 6.1.3: Growth Opportunities for the Global Mucopolysaccharidosis Treatment Market by End Use
  • 6.1.4: Growth Opportunities for the Global Mucopolysaccharidosis Treatment Market by Region
• 6.2: Emerging Trends in the Global Mucopolysaccharidosis Treatment Market
• 6.3: Strategic Analysis
  • 6.3.1: New Product Development
  • 6.3.2: Capacity Expansion of the Global Mucopolysaccharidosis Treatment Market
  • 6.3.3: Mergers, Acquisitions, and Joint Ventures in the Global Mucopolysaccharidosis Treatment Market
  • 6.3.4: Certification and Licensing

7. Company Profiles of Leading Players

• 7.1: Take da Pharmaceutical
• 7.2: BioMarin Pharmaceuticals
• 7.3: Ultragenyx Pharmaceutical
• 7.4: Sarepta Therapeutics
• 7.5: Abeona Therapeutics
• 7.6: Eloxx Pharmaceuticals
• 7.7: Esteve
• 7.8: Immusoft Corporation
• 7.9: Inventiva
• 7.10: GC Pharma