Lysosomal Storage Disease Treatment Market Forecast, 2034 

Lysosomal Storage Disease Treatment Market - By Treatment Type, By Disease Type, By Age Group, By End Use - Global Forecast, 2025-2034

Report Content

Chapter 1&nbsp;&nbsp;&nbsp;Methodology and Scope

1.1&nbsp;&nbsp;&nbsp; Market scope and definition

1.2&nbsp;&nbsp;&nbsp; Research design

1.2.1&nbsp;&nbsp;&nbsp; Research approach

1.2.2&nbsp;&nbsp;&nbsp; Data collection methods

1.3&nbsp;&nbsp;&nbsp; Data mining sources

1.3.1&nbsp;&nbsp;&nbsp; Global

1.3.2&nbsp;&nbsp;&nbsp; Regional/Country

1.4&nbsp;&nbsp;&nbsp; Base estimates and calculations

1.4.1&nbsp;&nbsp;&nbsp; Base year calculation

1.4.2&nbsp;&nbsp;&nbsp; Key trends for market estimation

1.5&nbsp;&nbsp;&nbsp; Primary research and validation

1.5.1&nbsp;&nbsp;&nbsp; Primary sources

1.6&nbsp;&nbsp;&nbsp; Forecast model

1.7&nbsp;&nbsp;&nbsp; Research assumptions and limitations

Chapter 2&nbsp;&nbsp;&nbsp;Executive Summary

2.1&nbsp;&nbsp;&nbsp; Industry 3600&nbsp;synopsis

2.2&nbsp;&nbsp;&nbsp; Key market trends

2.2.1&nbsp;&nbsp;&nbsp; Regional trends

2.2.2&nbsp;&nbsp;&nbsp; Treatment type trends

2.2.3&nbsp;&nbsp;&nbsp; Disease type trends

2.2.4&nbsp;&nbsp;&nbsp; Age group trends

2.2.5&nbsp;&nbsp;&nbsp; End use trends

2.3&nbsp;&nbsp;&nbsp; CXO perspectives: Strategic imperatives

2.3.1&nbsp;&nbsp;&nbsp; Key decision points for industry executives

2.3.2&nbsp;&nbsp;&nbsp; Critical success factors for market players

2.4&nbsp;&nbsp;&nbsp; Future outlook and strategic recommendations

Chapter 3&nbsp;&nbsp;&nbsp;Industry Insights

3.1&nbsp;&nbsp;&nbsp; Industry ecosystem analysis

3.2&nbsp;&nbsp;&nbsp; Industry impact forces

3.2.1&nbsp;&nbsp;&nbsp; Growth drivers

3.2.1.1&nbsp;&nbsp;&nbsp; Increasing diagnosis due to newborn-screening programs

3.2.1.2&nbsp;&nbsp;&nbsp; Growing pipeline of gene and enzyme therapies

3.2.1.3&nbsp;&nbsp;&nbsp; Advancement in molecular and biomarker-based diagnosis

3.2.1.4&nbsp;&nbsp;&nbsp; Increasing shift towards disease-modifying and curative therapies

3.2.2&nbsp;&nbsp;&nbsp; Industry pitfalls and challenges

3.2.2.1&nbsp;&nbsp;&nbsp; High cost of treatment

3.2.2.2&nbsp;&nbsp;&nbsp; Limited penetration in low-and middle-income countries (LMICs)

3.2.3&nbsp;&nbsp;&nbsp; Market opportunities

3.2.3.1&nbsp;&nbsp;&nbsp; Growing demand of gene therapy and genome editing

3.2.3.2&nbsp;&nbsp;&nbsp; Rise in specialized rare disease treatment centers

3.3&nbsp;&nbsp;&nbsp; Growth potential analysis

3.4&nbsp;&nbsp;&nbsp; Regulatory landscape

3.4.1&nbsp;&nbsp;&nbsp; North America

3.4.2&nbsp;&nbsp;&nbsp; Europe

3.4.3&nbsp;&nbsp;&nbsp; Asia Pacific

3.4.4&nbsp;&nbsp;&nbsp; Latin America

3.4.5&nbsp;&nbsp;&nbsp; Middle East and Africa

3.5&nbsp;&nbsp;&nbsp; Technology landscape

3.5.1&nbsp;&nbsp;&nbsp; Current technological trends

3.5.2&nbsp;&nbsp;&nbsp; Emerging technologies

3.6&nbsp;&nbsp;&nbsp; Future market trends

3.7&nbsp;&nbsp;&nbsp; Pricing analysis

3.8&nbsp;&nbsp;&nbsp; Porter&rsquo;s analysis

3.9&nbsp;&nbsp;&nbsp; PESTEL analysis

Chapter 4&nbsp;&nbsp;&nbsp;Competitive Landscape, 2024

4.1&nbsp;&nbsp;&nbsp; Introduction

4.2&nbsp;&nbsp;&nbsp; Company market share analysis

4.3&nbsp;&nbsp;&nbsp; Company matrix analysis

4.4&nbsp;&nbsp;&nbsp; Competitive analysis of major market players

4.5&nbsp;&nbsp;&nbsp; Competitive positioning matrix

4.6&nbsp;&nbsp;&nbsp; Key developments

4.6.1&nbsp;&nbsp;&nbsp; Merger and acquisition

4.6.2&nbsp;&nbsp;&nbsp; Partnership and collaboration

4.6.3&nbsp;&nbsp;&nbsp; New product launches

4.6.4&nbsp;&nbsp;&nbsp; Expansion plans

Chapter 5&nbsp;&nbsp;&nbsp;Market Estimates and Forecast, By Treatment Type, 2021 - 2034 ($ Mn)

5.1&nbsp;&nbsp;&nbsp; Key trends

5.2&nbsp;&nbsp;&nbsp; Enzyme replacement therapy

5.3&nbsp;&nbsp;&nbsp; Stem cell transplants

5.4&nbsp;&nbsp;&nbsp; Substrate reduction therapy

5.5&nbsp;&nbsp;&nbsp; Other treatment types

Chapter 6&nbsp;&nbsp;&nbsp;Market Estimates and Forecast, By Disease Type, 2021 - 2034 ($ Mn)

6.1&nbsp;&nbsp;&nbsp; Key trends

6.2&nbsp;&nbsp;&nbsp; Gaucher disease

6.3&nbsp;&nbsp;&nbsp; Mucopolysaccharidoses

6.4&nbsp;&nbsp;&nbsp; Pompe disease

6.5&nbsp;&nbsp;&nbsp; Fabry disease

6.6&nbsp;&nbsp;&nbsp; Other disease types

Chapter 7&nbsp;&nbsp;&nbsp;Market Estimates and Forecast, By Age Group, 2021 - 2034 ($ Mn)

7.1&nbsp;&nbsp;&nbsp; Key trends

7.2&nbsp;&nbsp;&nbsp; Pediatric

7.3&nbsp;&nbsp;&nbsp; Adult

Chapter 8&nbsp;&nbsp;&nbsp;Market Estimates and Forecast, By End Use, 2021 - 2034 ($ Mn)

8.1&nbsp;&nbsp;&nbsp; Key trends

8.2&nbsp;&nbsp;&nbsp; Hospitals

8.3&nbsp;&nbsp;&nbsp; Specialty clinics

8.4&nbsp;&nbsp;&nbsp; Homecare settings

8.5&nbsp;&nbsp;&nbsp; Other end use

Chapter 9&nbsp;&nbsp;&nbsp;Market Estimates and Forecast, By Region, 2021 - 2034 ($ Mn)

9.1&nbsp;&nbsp;&nbsp; Key trends

9.2&nbsp;&nbsp;&nbsp; North America

9.2.1&nbsp;&nbsp;&nbsp; U.S.

9.2.2&nbsp;&nbsp;&nbsp; Canada

9.3&nbsp;&nbsp;&nbsp; Europe

9.3.1&nbsp;&nbsp;&nbsp; Germany

9.3.2&nbsp;&nbsp;&nbsp; UK

9.3.3&nbsp;&nbsp;&nbsp; France

9.3.4&nbsp;&nbsp;&nbsp; Spain

9.3.5&nbsp;&nbsp;&nbsp; Italy

9.3.6&nbsp;&nbsp;&nbsp; Netherlands

9.4&nbsp;&nbsp;&nbsp; Asia Pacific

9.4.1&nbsp;&nbsp;&nbsp; China

9.4.2&nbsp;&nbsp;&nbsp; Japan

9.4.3&nbsp;&nbsp;&nbsp; India

9.4.4&nbsp;&nbsp;&nbsp; Australia

9.4.5&nbsp;&nbsp;&nbsp; South Korea

9.5&nbsp;&nbsp;&nbsp; Latin America

9.5.1&nbsp;&nbsp;&nbsp; Brazil

9.5.2&nbsp;&nbsp;&nbsp; Mexico

9.5.3&nbsp;&nbsp;&nbsp; Argentina

9.6&nbsp;&nbsp;&nbsp; Middle East and Africa

9.6.1&nbsp;&nbsp;&nbsp; South Africa

9.6.2&nbsp;&nbsp;&nbsp; Saudi Arabia

9.6.3&nbsp;&nbsp;&nbsp; UAE

Chapter 10&nbsp;&nbsp;&nbsp;Company Profiles

10.1&nbsp;&nbsp;&nbsp; Alexion Pharmaceuticals

10.2&nbsp;&nbsp;&nbsp; Amicus Therapeutics

10.3&nbsp;&nbsp;&nbsp; Avrobio

10.4&nbsp;&nbsp;&nbsp; BioMarin

10.5&nbsp;&nbsp;&nbsp; JCR Pharmaceuticals

10.6&nbsp;&nbsp;&nbsp; Johnson &amp; Johnson (Actelion Pharmaceuticals)

10.7&nbsp;&nbsp;&nbsp; Orchard Therapeutics

10.8&nbsp;&nbsp;&nbsp; Orphazyme

10.9&nbsp;&nbsp;&nbsp; Pfizer

10.10&nbsp;&nbsp;&nbsp;Sanofi (Genzyme Corporation)

10.11&nbsp;&nbsp;&nbsp;Sigilon Therapeutics

10.12&nbsp;&nbsp;&nbsp;Sangamo therapeutics

10.13&nbsp;&nbsp;&nbsp;Takeda Pharmaceutical Company (Shire)

Lysosomal Storage Disease Treatment Market

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