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Global Duchenne Muscular Dystrophy Drugs Market by Drug Type, by Distribution Channel, and By Region

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    Report

  • 200 Pages
  • March 2024
  • Region: Global
  • Erevna Healthcare
  • ID: 5941910
The global Duchenne muscular dystrophy (DMD) drugs market size was estimated to be USD 2.96 billion in 2023 and is expected to reach USD 111.36 billion by 2034 with a CAGR of 39.12% during the forecast period 2024-2034. The increasing prevalence of Duchenne muscular dystrophy, advancements in genetic research, gene therapy, and genetic editing technologies, government initiatives and support, increased collaboration and partnerships, and technological advancements in drug delivery will drive market growth.

Finding viable treatments and therapies has been a major driving force behind research and development efforts due to the increasing frequency of DMD. Novel therapeutic approaches for genetic diseases such as DMD have become possible with the advent of gene therapy and genetic editing technology. For instance, in October 2023, ambulatory boys with Duchenne mutations susceptible to skipping exons 45-50 had good preliminary findings from the Phase 3 PROMINENCE trial assessing SRP-9041, a next-generation exon skipping option.

By drug type, the corticosteroids segment accounted for the highest revenue-grossing segment in the global Duchenne muscular dystrophy (DMD) drugs market in 2023 owing to the significant efficacy demonstrated in managing inflammation and delaying disease progression, along with the established track record of corticosteroids, such as prednisone and deflazacort, as a standard-of-care treatment for Duchenne Muscular Dystrophy (DMD). For instance, PTC Therapeutics announced in November 2023 the start of the Phase 2b PULSAR project, which will assess PTC518-a next-generation nonsense mutation treatment candidate-in conjunction with Ataluren in patients with Duchenne who have nonsense mutations. Additionally, the exon-skipping therapies segment is predicted to grow at the fastest CAGR during the forecast period owing to the advancements in precision medicine, significant research and development investments, and the increasing adoption of exon-skipping therapies, such as Eteplirsen, Golodirsen, and Viltolarsen, which have demonstrated promising outcomes in treating specific genetic mutations associated with Duchenne Muscular Dystrophy (DMD).

By distribution channel, the hospital's segment accounted for the highest revenue-grossing segment in the global Duchenne muscular dystrophy (DMD) drugs market in 2023 owing to the established infrastructure and specialized medical facilities available in hospitals, facilitating the diagnosis, treatment, and management of Duchenne Muscular Dystrophy (DMD). For instance, Pfizer Inc. presented encouraging results from the Phase 3 DELOS investigation in October 2023, indicating that Vyondys 53 continues to be safe and effective in ambulatory boys with Duchenne mutations who are able to skip exons 53-55. Additionally, the specialty pharmacies segment is predicted to grow at the fastest CAGR during the forecast period owing to the increasing emphasis on personalized patient care, improved accessibility to orphan drugs, and the rising trend of home-based treatments for Duchenne Muscular Dystrophy (DMD).

North American region is anticipated to have the highest revenue share during the forecast period owing to the robust healthcare infrastructure, extensive research and development activities, and a well-established regulatory framework for drug approvals. Additionally, the Asia Pacific region is predicted to grow at the fastest CAGR during the forecast period owing to the increasing awareness of Duchenne Muscular Dystrophy (DMD), a growing patient population, and rising investments in healthcare infrastructure and research and development. For instance, in September 2023, the European Commission permitted Santhera Pharmaceuticals to commercialize idebenone to treat muscular weakness in ambulatory Duchenne patients six years of age and older.

This comprehensive research report focuses on the global and regional market size and forecasts from 2023 to 2034.

Report Scope:

  • Base Year: 2023
  • Forecast Period: 2024-2034
  • Study Coverage
  • Market Forecast by Drug Type, and Distribution Channel
  • Market Forecast for 5 Regions and 17+ Countries
  • North America (U.S. and Canada)
  • Europe (Germany, France, UK, Spain, Italy, Russia, Rest of Europe)
  • Asia Pacific (China, Japan, India, Australia, South Korea, Rest of APAC)
  • Latin America (Brazil, Mexico, Argentina, Rest of LATAM)
  • MEA (South Africa, GCC, Rest of MEA)
  • Exhaustive Company Profiles of the Top 10+ Major Market Players
  • 20% Free Customization Available to Meet Your Exact Requirement.

Segmentation: Duchenne Muscular Dystrophy (DMD) Drugs Market Report 2023 - 2034

Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis & Forecast by Drug Type 2023 - 2034 (Revenue USD Bn)

  • Corticosteroids
  • Exon Skipping Therapies
  • Nonsense Mutation Therapies
  • Gene Therapy
  • Myostatin Inhibitors

Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis & Forecast by Distribution Channel 2023 - 2034 (Revenue USD Bn)

  • Hospitals
  • Specialty Pharmacies
  • Others

Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis & Forecast by Region 2023 - 2034 (Revenue USD Bn)

  • North America
  • U.S.
  • Canada
  • Europe
  • Germany
  • France
  • UK
  • Spain
  • Italy
  • Rest of Europe
  • Asia Pacific
  • China
  • Japan
  • India
  • Australia
  • South Korea
  • Rest of APAC
  • Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of LATAM
  • Middle East & Africa
  • South Africa
  • GCC
  • Rest of MEA

Table of Contents

1. Research Methodology
1.1. Study Objectives
1.2. Study Scope
1.3. Research Assumptions
1.4. Research Framework
2. Introduction
2.1. Market Definition
2.2. Global Duchenne Muscular Dystrophy (DMD) Drugs Market Overview
3. Executive Summary
4. Market Environment Analysis
4.1. Porter’s 5 Forces Analysis
4.2. PESTEL Analysis
4.3. SWOT Analysis
5. Market Dynamics
5.1. Drivers Analysis
5.2. Restraints Analysis
5.3. Opportunities Analysis
5.4. Threats Analysis
5.5. Trend Analysis
6. COVID-19 Impact Analysis
7. Duchenne Muscular Dystrophy (DMD) Drugs Market: Drug Type Estimates & Trend Analysis
7.1. Drug Type Segment Opportunity Analysis
7.2. Corticosteroids
7.2.1. Corticosteroids Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
7.3. Exon Skipping Therapies
7.3.1. Exon Skipping Therapies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
7.4. Nonsense Mutation Therapies
7.4.1. Nonsense Mutation Therapies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
7.5. Gene Therapy
7.5.1. Gene Therapy Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
7.6. Myostatin Inhibitors
7.6.1. Myostatin Inhibitors Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8. Duchenne Muscular Dystrophy (DMD) Drugs Market: Distribution Channel Estimates & Trend Analysis
8.1. Distribution Channel Segment Opportunity Analysis
8.2. Hospitals
8.2.1. Hospitals Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.3. Specialty Pharmacies
8.3.1. Specialty Pharmacies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.4. Others
8.4.1. Others Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
9. Regional Market Analysis
9.1. Regional Market Opportunity Analysis
10. North America Duchenne Muscular Dystrophy (DMD) Drugs Market
10.1. North America Duchenne Muscular Dystrophy (DMD) Drugs Market
10.1.1. North America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
10.1.2. North America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
10.1.3. North America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
10.1.4. North America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
10.2. U.S. Global Duchenne Muscular Dystrophy (DMD) Drugs Market
10.2.1. U.S. Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
10.2.2. U.S. Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
10.2.3. U.S. Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
10.3. Canada Global Duchenne Muscular Dystrophy (DMD) Drugs Market
10.3.1. Canada Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
10.3.2. Canada Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
10.3.3. Canada Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11. Europe Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.1. Europe Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.1.1. Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.1.2. Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
11.1.3. Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.1.4. Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.2. Germany Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.2.1. Germany Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.2.2. Germany Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.2.3. Germany Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.3. UK Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.3.1. UK Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.3.2. UK Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.3.3. UK Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.4. France Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.4.1. France Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.4.2. France Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.4.3. France Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.5. Spain Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.5.1. Spain Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.5.2. Spain Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.5.3. Spain Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.6. Italy Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.6.1. Italy Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.6.2. Italy Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.6.3. Italy Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.7. Rest of Europe Global Duchenne Muscular Dystrophy (DMD) Drugs Market
11.7.1. Rest of Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.7.2. Rest of Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.7.3. Rest of Europe Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12. Asia Pacific Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.1. Asia Pacific Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.1.1. Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.1.2. Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
12.1.3. Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.1.4. Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.2. Japan Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.2.1. Japan Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.2.2. Japan Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.2.3. Japan Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.3. China Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.3.1. China Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.3.2. China Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.3.3. China Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.4. India Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.4.1. India Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.4.2. India Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.4.3. India Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.5. South Korea Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.5.1. South Korea Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.5.2. South Korea Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.5.3. South Korea Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.6. Australia Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.6.1. Australia Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.6.2. Australia Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.6.3. Australia Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.7. Rest of Asia Pacific Global Duchenne Muscular Dystrophy (DMD) Drugs Market
12.7.1. Rest of Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.7.2. Rest of Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.7.3. Rest of Asia Pacific Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13. Latin America Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.1. Latin America Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.1.1. Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.1.2. Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
13.1.3. Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.1.4. Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.2. Brazil Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.2.1. Brazil Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.2.2. Brazil Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.2.3. Brazil Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.3. Mexico Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.3.1. Mexico Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.3.2. Mexico Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.3.3. Mexico Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.4. Argentina Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.4.1. Argentina Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.4.2. Argentina Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.4.3. Argentina Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.5. Rest of Latin America Global Duchenne Muscular Dystrophy (DMD) Drugs Market
13.5.1. Rest of Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.5.2. Rest of Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.5.3. Rest of Latin America Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14. MEA Global Duchenne Muscular Dystrophy (DMD) Drugs Market
14.1. MEA Global Duchenne Muscular Dystrophy (DMD) Drugs Market
14.1.1. MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.1.2. MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
14.1.3. MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.1.4. MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.2. GCC Global Duchenne Muscular Dystrophy (DMD) Drugs Market
14.2.1. GCC Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.2.2. GCC Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.2.3. GCC Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.3. South Africa Global Duchenne Muscular Dystrophy (DMD) Drugs Market
14.3.1. South Africa Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.3.2. South Africa Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.3.3. South Africa Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.4. Rest of MEA Global Duchenne Muscular Dystrophy (DMD) Drugs Market
14.4.1. Rest of MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.4.2. Rest of MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.4.3. Rest of MEA Duchenne Muscular Dystrophy (DMD) Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
15. Competitor Analysis
15.1. Company Market Share Analysis, 2023
15.2. Major Recent Developments
16. Company Profiles
16.1. Sarepta Therapeutics
16.2. Pfizer Inc.
16.3. Santhera Pharmaceuticals
16.4. PTC Therapeutics
16.5. Summit Therapeutics
16.6. Catabasis Pharmaceuticals
16.7. CureDuchenne
16.8. Capricor Therapeutics
16.9. Wave Life Sciences
16.10. NS Pharma (Nippon Shinyaku)
16.11. Solid Biosciences
16.12. Beech Tree Labs
16.13. Roche (Genentech)
16.14. Avidity Biosciences
16.15. Acceleron Pharma
17. Conclusion18. Recommendations

Companies Mentioned

  • Sarepta Therapeutics
  • Pfizer Inc.
  • Santhera Pharmaceuticals
  • PTC Therapeutics
  • Summit Therapeutics
  • Catabasis Pharmaceuticals
  • CureDuchenne
  • Capricor Therapeutics
  • Wave Life Sciences
  • NS Pharma (Nippon Shinyaku)
  • Solid Biosciences
  • Beech Tree Labs
  • Roche (Genentech)
  • Avidity Biosciences
  • Acceleron Pharma.

Table Information