세계의 리소좀 축적 질환 치료 시장 - 규모, 점유율, 동향, 산업 분석 보고서 : 질환 유형별, 치료 유형별, 최종 사용자별, 지역별 시장 예측(2025-2034년)

The lysosomal storage disease treatment market size is expected to reach USD 6.80 Billion by 2034, according to a new study by Polaris Market Research. The report "Lysosomal Storage Disease Treatment Market Size, Share, Trends, & Industry Analysis Report By Disease Type (Gaucher's Diseases, Fabry Diseases, Pompe's Syndrome, Mucopolysaccharidosis, and Others), By Type of Therapy, By End User, and By Region - Market Forecast, 2025-2034" gives a detailed insight into current market dynamics and provides analysis on future market growth.

The lysosomal storage disease (LSD) treatment market is witnessing steady progress as pharmaceutical companies and research institutions develop new therapies aimed at addressing rare genetic disorders. Growing awareness, increasing patient advocacy, and supportive regulatory frameworks are helping accelerate drug approvals and improve access to innovative treatments.

Rising investments in biotechnology and personalized medicine are shifting the market focus beyond symptom control toward long-term disease management and the development of potential cures. This evolution reflects a strong push toward improving patient outcomes and delivering more effective, targeted therapies for conditions such as Gaucher disease, Fabry disease, and Pompe disease.

Lysosomal Storage Disease Treatment Market Report Highlights

Based on disease type, gaucher's disease segment dominated the market in 2024, driven by its wider global prevalence and the presence of an established therapeutic ecosystem.

In terms of type of therapy, the enzyme replacement therapy (ERT) segment dominated the market in 2024. This is driven by the increasing adoption in managing various lysosomal storage disorders.

In terms of end user, the hospital segment dominated the market in 2024, attributed to delivery of specialized interventions such as enzyme replacement therapies and stem cell-based procedures.

North America accounted for the majority share of 36.98 in the global lysosomal storage disease treatment market in 2024, due to the strong presence of biotech and pharmaceutical firms advancing treatments for rare genetic conditions.

Asia Pacific held 6.13% market share, owing to ongoing improvements in healthcare infrastructure and access to advanced diagnostic capabilities.

A few global key market players include Alexion Pharmaceuticals, Inc., Amicus Therapeutics, Inc., BioMarin Pharmaceutical Inc., Eli Lilly and Company, Johnson & Johnson (Actelion Pharmaceuticals Ltd.), Merck & Co., Inc., Novartis AG, Pfizer Inc., Sanofi (Genzyme Corporation), Sigilon Therapeutics, Inc., Takeda Pharmaceutical Company Limited (Shire Plc), Chiesi Farmaceutici S.p.A., Orphazyme A/S, Protalix BioTherapeutics, Inc., and Ultragenyx Pharmaceutical Inc.

Polaris Market Research has segmented the market report on the basis of disease type, type of therapy, end user, and region:

By Disease Type Outlook (Revenue, USD Billion, 2020-2034)

Gaucher's Diseases

Fabry Diseases

Pompe's Syndrome

Mucopolysaccharidosis

Others

By Type of Therapy Outlook (Revenue, USD Billion, 2020-2034)

Enzyme Replacement Therapy

Stem Cell Therapy

Substrate Reduction Therapy

Others

By End User Outlook (Revenue, USD Billion, 2020-2034)

Hospitals

Clinics

Others

By Regional Outlook (Revenue, USD Billion, 2020-2034)

North America

US

Canada

Europe

Germany

France

UK

Italy

Spain

Netherlands

Russia

Rest of Europe

Asia Pacific

China

Japan

India

Malaysia

South Korea

Indonesia

Australia

Vietnam

Rest of Asia Pacific

Middle East & Africa

Saudi Arabia

UAE

Israel

South Africa

Rest of Middle East & Africa

Latin America

Mexico

Brazil

Argentina

Rest of Latin America

Table of Contents

1. Introduction

• 1.1. Report Description
  • 1.1.1. Objectives of the Study
  • 1.1.2. Market Scope
  • 1.1.3. Assumptions
• 1.2. Stakeholders

2. Executive Summary

• 2.1. Market Highlights

3. Research Methodology

• 3.1. Overview
  • 3.1.1. Data Mining
• 3.2. Data Sources
  • 3.2.1. Primary Sources
  • 3.2.2. Secondary Sources

4. Global Lysosomal Storage Disease Treatment Market Insights

• 4.1. Lysosomal Storage Disease Treatment Market - Market Snapshot
• 4.2. Lysosomal Storage Disease Treatment Market Dynamics
  • 4.2.1. Drivers and Opportunities
    • 4.2.1.1. Rising Incidence of Rare Genetic Disorders
    • 4.2.1.2. Advancements in Enzyme Replacement Therapy (ERT)
  • 4.2.2. Restraints and Challenges
    • 4.2.2.1. High Treatment Costs and Limited Reimbursement
• 4.3. Porter's Five Forces Analysis
  • 4.3.1. Bargaining Power of Suppliers (Moderate)
  • 4.3.2. Threats of New Entrants: (Low)
  • 4.3.3. Bargaining Power of Buyers (Moderate)
  • 4.3.4. Threat of Substitute (Moderate)
  • 4.3.5. Rivalry among existing firms (High)
• 4.4. PESTEL Analysis
• 4.5. Lysosomal Storage Disease Treatment Market Trends
• 4.6. Value Chain Analysis

5. Global Lysosomal Storage Disease Treatment Market, by Disease Type

• 5.1. Key Findings
• 5.2. Introduction
  • 5.2.1. Global Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
• 5.3. Gaucher's Diseases
  • 5.3.1. Global Lysosomal Storage Disease Treatment Market, by Gaucher's Diseases, by Region, 2020-2034 (USD Billion)
• 5.4. Fabry Diseases
  • 5.4.1. Global Lysosomal Storage Disease Treatment Market, by Fabry Diseases, by Region, 2020-2034 (USD Billion)
• 5.5. Pompe's Syndrome
  • 5.5.1. Global Lysosomal Storage Disease Treatment Market, by Pompe's Syndrome, by Region, 2020-2034 (USD Billion)
• 5.6. Mucopolysaccharidosis
  • 5.6.1. Global Lysosomal Storage Disease Treatment Market, by Mucopolysaccharidosis, by Region, 2020-2034 (USD Billion)
• 5.7. Others
  • 5.7.1. Global Lysosomal Storage Disease Treatment Market, by Others, by Region, 2020-2034 (USD Billion)

6. Global Lysosomal Storage Disease Treatment Market, by Type of Therapy

• 6.1. Key Findings
• 6.2. Introduction
  • 6.2.1. Global Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
• 6.3. Enzyme Replacement Therapy
  • 6.3.1. Global Lysosomal Storage Disease Treatment Market, by Enzyme Replacement Therapy, by Region, 2020-2034 (USD Billion)
• 6.4. Stem Cell Therapy
  • 6.4.1. Global Lysosomal Storage Disease Treatment Market, by Stem Cell Therapy, by Region, 2020-2034 (USD Billion)
• 6.5. Substrate Reduction Therapy
  • 6.5.1. Global Lysosomal Storage Disease Treatment Market, by Substrate Reduction Therapy, by Region, 2020-2034 (USD Billion)
• 6.6. Others
  • 6.6.1. Global Lysosomal Storage Disease Treatment Market, by Others, by Region, 2020-2034 (USD Billion)

7. Global Lysosomal Storage Disease Treatment Market, by End User

• 7.1. Key Findings
• 7.2. Introduction
  • 7.2.1. Global Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
• 7.3. Hospitals
  • 7.3.1. Global Lysosomal Storage Disease Treatment Market, by Hospitals, by Region, 2020-2034 (USD Billion)
• 7.4. Clinics
  • 7.4.1. Global Lysosomal Storage Disease Treatment Market, by Clinics, by Region, 2020-2034 (USD Billion)
• 7.5. Others
  • 7.5.1. Global Lysosomal Storage Disease Treatment Market, by Others, by Region, 2020-2034 (USD Billion)

8. Global Lysosomal Storage Disease Treatment Market, by Geography

• 8.1. Key Findings
• 8.2. Introduction
  • 8.2.1. Lysosomal Storage Disease Treatment Market Assessment, By Geography, 2020-2034 (USD Billion)
• 8.3. Lysosomal Storage Disease Treatment Market - North America
  • 8.3.1. North America: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
  • 8.3.2. North America: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
  • 8.3.3. North America: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.3.4. Lysosomal Storage Disease Treatment Market - U.S.
    • 8.3.4.1. U.S.: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.3.4.2. U.S.: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.3.4.3. U.S.: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.3.5. Lysosomal Storage Disease Treatment Market - Canada
    • 8.3.5.1. Canada: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.3.5.2. Canada: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.3.5.3. Canada: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
• 8.4. Lysosomal Storage Disease Treatment Market - Europe
  • 8.4.1. Europe: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
  • 8.4.2. Europe: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
  • 8.4.3. Europe: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.4. Lysosomal Storage Disease Treatment Market - UK
    • 8.4.4.1. UK: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.4.2. UK: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.4.3. UK: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.5. Lysosomal Storage Disease Treatment Market - France
    • 8.4.5.1. France: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.5.2. France: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.5.3. France: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.6. Lysosomal Storage Disease Treatment Market - Germany
    • 8.4.6.1. Germany: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.6.2. Germany: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.6.3. Germany: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.7. Lysosomal Storage Disease Treatment Market - Italy
    • 8.4.7.1. Italy: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.7.2. Italy: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.7.3. Italy: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.8. Lysosomal Storage Disease Treatment Market - Spain
    • 8.4.8.1. Spain: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.8.2. Spain: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.8.3. Spain: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.9. Lysosomal Storage Disease Treatment Market - Netherlands
    • 8.4.9.1. Netherlands: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.9.2. Netherlands: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.9.3. Netherlands: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.10. Lysosomal Storage Disease Treatment Market - Russia
    • 8.4.10.1. Russia: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.10.2. Russia: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.10.3. Russia: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.4.11. Lysosomal Storage Disease Treatment Market - Rest of Europe
    • 8.4.11.1. Rest of Europe: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.4.11.2. Rest of Europe: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.4.11.3. Rest of Europe: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
• 8.5. Lysosomal Storage Disease Treatment Market - Asia Pacific
  • 8.5.1. Asia Pacific: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
  • 8.5.2. Asia Pacific: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
  • 8.5.3. Asia Pacific: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.4. Lysosomal Storage Disease Treatment Market - China
    • 8.5.4.1. China: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.4.2. China: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.4.3. China: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.5. Lysosomal Storage Disease Treatment Market - India
    • 8.5.5.1. India: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.5.2. India: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.5.3. India: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.6. Lysosomal Storage Disease Treatment Market - Malaysia
    • 8.5.6.1. Malaysia: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.6.2. Malaysia: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.6.3. Malaysia: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.7. Lysosomal Storage Disease Treatment Market - Japan
    • 8.5.7.1. Japan: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.7.2. Japan: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.7.3. Japan: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.8. Lysosomal Storage Disease Treatment Market - Indonesia
    • 8.5.8.1. Indonesia: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.8.2. Indonesia: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.8.3. Indonesia: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.9. Lysosomal Storage Disease Treatment Market - South Korea
    • 8.5.9.1. South Korea: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.9.2. South Korea: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.9.3. South Korea: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.10. Lysosomal Storage Disease Treatment Market - Australia
    • 8.5.10.1. Australia: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.10.2. Australia: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.10.3. Australia: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.5.11. Lysosomal Storage Disease Treatment Market - Rest of Asia Pacific
    • 8.5.11.1. Rest of Asia Pacific: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.5.11.2. Rest of Asia Pacific: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.5.11.3. Rest of Asia Pacific: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
• 8.6. Lysosomal Storage Disease Treatment Market - Middle East & Africa
  • 8.6.1. Middle East & Africa: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
  • 8.6.2. Middle East & Africa: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
  • 8.6.3. Middle East & Africa: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.6.4. Lysosomal Storage Disease Treatment Market - Saudi Arabia
    • 8.6.4.1. Saudi Arabia: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.6.4.2. Saudi Arabia: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.6.4.3. Saudi Arabia: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.6.5. Lysosomal Storage Disease Treatment Market - UAE
    • 8.6.5.1. UAE: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.6.5.2. UAE: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.6.5.3. UAE: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.6.6. Lysosomal Storage Disease Treatment Market - Israel
    • 8.6.6.1. Israel: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.6.6.2. Israel: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.6.6.3. Israel: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.6.7. Lysosomal Storage Disease Treatment Market - South Africa
    • 8.6.7.1. South Africa: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.6.7.2. South Africa: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.6.7.3. South Africa: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.6.8. Lysosomal Storage Disease Treatment Market - Rest of Middle East & Africa
    • 8.6.8.1. Rest of Middle East & Africa: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.6.8.2. Rest of Middle East & Africa: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.6.8.3. Rest of Middle East & Africa: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
• 8.7. Lysosomal Storage Disease Treatment Market - Latin America
  • 8.7.1. Latin America: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
  • 8.7.2. Latin America: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
  • 8.7.3. Latin America: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.7.4. Lysosomal Storage Disease Treatment Market - Mexico
    • 8.7.4.1. Mexico: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.7.4.2. Mexico: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.7.4.3. Mexico: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.7.5. Lysosomal Storage Disease Treatment Market - Brazil
    • 8.7.5.1. Brazil: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.7.5.2. Brazil: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.7.5.3. Brazil: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.7.6. Lysosomal Storage Disease Treatment Market - Argentina
    • 8.7.6.1. Argentina: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.7.6.2. Argentina: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.7.6.3. Argentina: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)
  • 8.7.7. Lysosomal Storage Disease Treatment Market - Rest of Latin America
    • 8.7.7.1. Rest of Latin America: Lysosomal Storage Disease Treatment Market, by Disease Type, 2020-2034 (USD Billion)
    • 8.7.7.2. Rest of Latin America: Lysosomal Storage Disease Treatment Market, by Type of Therapy, 2020-2034 (USD Billion)
    • 8.7.7.3. Rest of Latin America: Lysosomal Storage Disease Treatment Market, by End User, 2020-2034 (USD Billion)

9. Competitive Landscape

• 9.1. Expansion and Acquisition Analysis
  • 9.1.1. Expansion
  • 9.1.2. Acquisitions
• 9.2. Partnerships/Collaborations/Agreements/Exhibitions

10. Company Profiles

• 10.1. Alexion Pharmaceuticals, Inc.
  • 10.1.1. Company Overview
  • 10.1.2. Financial Performance
  • 10.1.3. Disease Type Benchmarking
  • 10.1.4. Recent Development
• 10.2. Amicus Therapeutics, Inc.
  • 10.2.1. Company Overview
  • 10.2.2. Financial Performance
  • 10.2.3. Disease Type Benchmarking
  • 10.2.4. Recent Development
• 10.3. BioMarin Pharmaceutical Inc.
  • 10.3.1. Company Overview
  • 10.3.2. Financial Performance
  • 10.3.3. Disease Type Benchmarking
  • 10.3.4. Recent Development
• 10.4. Eli Lilly and Company
  • 10.4.1. Company Overview
  • 10.4.2. Financial Performance
  • 10.4.3. Disease Type Benchmarking
  • 10.4.4. Recent Development
• 10.5. Johnson & Johnson (Actelion Pharmaceuticals Ltd.)
  • 10.5.1. Company Overview
  • 10.5.2. Financial Performance
  • 10.5.3. Disease Type Benchmarking
  • 10.5.4. Recent Development
• 10.6. Merck & Co., Inc.
  • 10.6.1. Company Overview
  • 10.6.2. Financial Performance
  • 10.6.3. Disease Type Benchmarking
  • 10.6.4. Recent Development
• 10.7. Novartis AG
  • 10.7.1. Company Overview
  • 10.7.2. Financial Performance
  • 10.7.3. Disease Type Benchmarking
  • 10.7.4. Recent Development
• 10.8. Pfizer Inc.
  • 10.8.1. Company Overview
  • 10.8.2. Financial Performance
  • 10.8.3. Disease Type Benchmarking
  • 10.8.4. Recent Development
• 10.9. Sanofi (Genzyme Corporation)
  • 10.9.1. Company Overview
  • 10.9.2. Financial Performance
  • 10.9.3. Disease Type Benchmarking
  • 10.9.4. Recent Development
• 10.10. Sigilon Therapeutics, Inc.
  • 10.10.1. Company Overview
  • 10.10.2. Financial Performance
  • 10.10.3. Disease Type Benchmarking
  • 10.10.4. Recent Development
• 10.11. Takeda Pharmaceutical Company Limited (Shire Plc)
  • 10.11.1. Company Overview
  • 10.11.2. Financial Performance
  • 10.11.3. Disease Type Benchmarking
  • 10.11.4. Recent Development
• 10.12. Chiesi Farmaceutici S.p.A.
  • 10.12.1. Company Overview
  • 10.12.2. Financial Performance
  • 10.12.3. Disease Type Benchmarking
  • 10.12.4. Recent Development