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Global Duchenne Muscular Dystrophy Therapeutics Market By Drug (Translarna, Emflaza, EXONDYS 51), By Therapeutic Approach (Steroid Therapy, Exon Skipping, Mutation Suppression) Forecast to 2022

Published: May, 2017 | Pages: 100 | Publisher: RNCOS
Industry: Healthcare | Report Format: Electronic (PDF)

Duchenne Muscular Dystrophy (DMD) is a fatal genetic disorders diagnosed in children around the world. DMD is often referred to as orphan or rare diseases, as it affects only a small portion of the population, approximately 1 in every 3,500 live male births. However, various treatment approaches are available now-a-days that can inhibit progression of the disease. Amongst, the most attractive are molecular based therapies, such as mutation suppression or exon skipping. Moreover, there are many more drugs in various phases of clinical trials, which will help in the growth of this market. 

According to RNCOS’ new research report “Global Duchenne Muscular Dystrophy Therapeutics Market By Drug (Translarna, Emflaza, EXONDYS 51), By Therapeutic Approach (Steroid Therapy, Exon Skipping, Mutation Suppression) Forecast to 2022”, the Duchenne Muscular Dystrophy Therapeutics industry can possibly turn into a multi-million dollar industry by the end of 2022, as new products, particularly those in the advanced stage of clinical studies or with pending approvals, may enter the market to boost the growth. 

The Duchenne Muscular Dystrophy Therapeutics market has very few marketed products, such as Emflaza, Translarna, and EXONDYS 51. The market is majorly in the research phase, from which most of its revenue is generated. Therefore, a major focus has been on the ongoing clinical trials for the development of innovative products. In this context, the study provides a comprehensive overview of various aspects of the clinical trials in the Duchenne Muscular Dystrophy market, such as phases, geographies, etc. The report also provides the sales of major marketed Duchenne Muscular Dystrophy products, and the list of the products in clinical/pre-clinical research along with their clinical phases.

The report provides a detailed analysis of the current and future market scenario of the global Duchenne Muscular Dystrophy Therapeutics market. RNCOS, in its report, further covers insight about the major drivers and challenges, along with the latest trends and developments impacting the industry growth. In addition, the report also highlights various opportunities available for growth of the global Duchenne Muscular Dystrophy Therapeutics market. The report also provides insights regarding the strategies adopted by the players from 2015 to 2017 for enhancing their market share. 
The segmentation of Duchenne Muscular Dystrophy Therapeutics market has been done on the basis of different therapeutic approaches and geographical regions. Primarily, the Duchenne Muscular Dystrophy Therapeutics market is dominated by mutation suppression and exon skipping approach, with several companies and academic institutions focusing on potential for each of these treatments. 

Based on the geography, the market is divided into three regions, namely - North America, Europe, and Asia Pacific. There are several treatments for DMD that are approved or under review in the European Union or are expected to be under review by regulatory agencies in the near future. This is a major reason for the dominant position of Europe in the Duchenne Muscular Dystrophy market. 

The later part of the report discusses some of the prominent players in the global Duchenne Muscular Dystrophy Therapeutics market. The market share analysis of these players is also provided in the report. Furthermore, a brief business overview of each player has been provided along with their business segments, product portfolios and recent developments. Overall, the report will prove as a complete source of knowledge and analysis for clients and potential investors.
 Table of Content

1. Analyst View 

2. Research Methodology

3. Duchenne Muscular Dystrophy (DMD): An Overview

4. Market Dynamics
    4.1 Market Drivers
          4.1.1 Growing Prevalence of Duchenne Muscular Dystrophy
          4.1.2 Rising Acceptance of Duchenne Muscular Dystrophy Drugs in the Market
          4.1.3 Increasing Funding Support & Grants
          4.1.4 Increasing Awareness
          4.1.5 Robust and Opportunistic Pipeline
     4.2 Restraints
          4.2.1 Pediatric Neuromuscular Disease
          4.2.2 Lack of Disease History and Research Participants
          4.2.3 Diagnosis and Management of Duchenne Muscular Dystrophy in Developing Countries
          4.2.4 High Cost of Care
     4.3 Opportunities
           4.3.1 PPMO: Next Generation Antisense Platform 
           4.3.2 Growing Market Opportunity in Emerging Countries
           4.3.3 High Unmet Need for an Effective Disease Modifying Therapy
           4.3.4 Gene Therapy
           4.3.5 Utrophin Modulation 
           4.3.6 Biomarkers for Duchenne Muscular Dystophy

5. Global Duchenne Muscular Dystrophy Therapeutics Market Outlook 2022

6. Approved Drugs
    6.1 Emflaza (deflazacort)
    6.2 EXONDYS 51 (eteplirsen)
    6.3 Translarna (ataluren)

7. Market Segmentation by Therapeutic Approach
    7.1 Mutation Suppression
    7.2 Steroid Therapy
    7.3 Exon Skipping Therapy

8. Market Segmentation by Geography
    8.1 Europe
    8.2 North America
    8.3 Asia Pacific

9. Clinical Trial Assessment & Pipeline Analysis
    9.1 Clinical Trials
          9.1.1 By Geography
          9.1.2 By Clinical Trial Phase
          9.1.3 By Key Players
    9.2 Pipeline Analysis

10. Industry Trends and Developments
     10.1 Administration of Corticosteroids
     10.2 Collaboration amongst Industry Players and Non-Industry Participants
     10.3 Exon-Skipping Technology Crowding the DMD Therapeutics Industry
     10.4 Carrier Screening for Duchenne Muscular Dystrophy

11. Strategic Alliances

12. Competitive Landscape

13. Key Player Analysis
     13.1 Marathon Pharmaceuticals, LLC
     13.2 PTC Therapeutics, Inc.
     13.3 Sarepta Therapeutics, Inc.
     13.4 ITALFARMACO S.p.A.
     13.5 Santhera Pharmaceuticals
     13.6 Bristol-Myers Squibb
     13.7 ReveraGen BioPharma, Inc.
     13.8 Catabasis Pharmaceuticals, Inc.
     13.9 FibroGen, Inc.
     13.10 NS Pharma, Inc.
     13.11 Pfizer
     13.12 Summit Therapeutics plc
     13.13 Taiho Pharmaceutical Co., Ltd. (Division of Otsuka Holdings Co. Ltd.)

14. Future Outlook
List of Tables:

Table 4-1: Grants for Duchenne Muscular Dystrophy
Table 4-2: Per-Patient Annual Costs of DMD, 2012
Table 6-1: Ongoing Trials for Emflaza
Table 6-2: Ongoing Trials for EXONDYS 51
Table 6-3: Ongoing Trials for Translarna
Table 7-1: Disease-Modifying Therapies for Duchenne Muscular Dystrophy
Table 9-1: Global Duchenne Muscular Dystrophy Therapeutics Pipeline
Table 13-1: Marathon Pharmaceuticals, LLC – Commercialized Duchenne Muscular Dystrophy Products
Table 13-2: PTC Therapeutics, Inc. - Key Financials (Million US$), 2014-2016
Table 13-3: PTC Therapeutics, Inc. – Commercialized Duchenne Muscular Dystrophy Products
Table 13-4: PTC Therapeutics, Inc. – Product Pipeline
Table 13-5: Sarepta Therapeutics, Inc. - Key Financials (Million US$), 2014-2016
Table 13-6: Sarepta Therapeutics, Inc. – Commercialized Duchenne Muscular Dystrophy Products
Table 13-7: Sarepta Therapeutics, Inc. – Product Pipeline
Table 13-8: ITALFARMACO S.p.A. – Product Pipeline
Table 13-9: Santhera Pharmaceuticals - Key Financials (Million US$), 2013-2015
Table 13-10: Santhera Pharmaceuticals – Product Pipeline
Table 13-11: Bristol Myers Squibb - Key Financials (Million US$), 2013-2015
Table 13-12: Bristol Myers Squibb – Product Pipeline
Table 13-13: ReveraGen BioPharma, Inc. – Product Pipeline
Table 13-14: Catabasis Pharmaceuticals, Inc. - Key Financials (Million US$), 2014-2016
Table 13-15: Catabasis Pharmaceuticals, Inc. – Product Pipeline
Table 13-16: FibroGen, Inc. - Key Financials (Million US$), 2014-2016
Table 13-17: FibroGen, Inc. – Product Pipeline
Table 13-18: NS Pharma, Inc. – Product Pipeline
Table 13-19: Pfizer Inc. - Key Financials (Million US$), 2014-2016
Table 13-20: Pfizer Inc. – Product Pipeline
Table 13-21: Summit Therapeutics plc – Product Pipeline
Table 13-22: Taiho Pharmaceutical Co., Ltd. - Key Financials (Million US$), 2014-2016 
Table 13-23: Taiho Pharmaceutical Co., Ltd. – Product Pipeline

List of Figures:

Figure 5-1: Global - Duchenne Muscular Dystrophy Therapeutics Market (Million US$), 2016-2022
Figure 6-1: Global - Sales of EXONDYS 51 (Million US$), 2016
Figure 6-2: Global - Sales of Translarna (Million US$), 2014-2017
Figure 7-1: Global - Duchenne Muscular Dystrophy Therapeutics Market by Therapeutic Approach (%), 2016
Figure 7-2: Global - Mutation Suppression Market for Duchenne Muscular Dystrophy (Million US$), 2016-2022
Figure 7-3: Global - Steroid Therapy Market for Duchenne Muscular Dystrophy (Million US$), 2016-2022
Figure 7-4: Global - Exon Skipping Therapy Market for Duchenne Muscular Dystrophy (Million US$), 2016-2022
Figure 8-1: Global - Duchenne Muscular Dystrophy Therapeutics Market by Geography (%), 2016
Figure 8-2: Europe - Duchenne Muscular Dystrophy Therapeutics Market (Million US$), 2016-2022
Figure 8-3: North America - Duchenne Muscular Dystrophy Therapeutics Market (Million US$), 2016-2022
Figure 8-4: Asia Pacific - Duchenne Muscular Dystrophy Therapeutics Market (Million US$), 2016-2022
Figure 9-1: Global - Duchenne Muscular Dystrophy Clinical Trials by Countries till April, 2017
Figure 9-2: Global - Duchenne Muscular Dystrophy Clinical Trials by Phase till April, 2017
Figure 9-3: Global - Duchenne Muscular Dystrophy Clinical Trials by Key Players till April, 2017
Figure 10-1: Exon Skipping Clinical Candidates
Figure 12-1: Global - Share of Major Players in Duchenne Muscular Dystrophy Therapeutics Market (%), 2016
Figure 13-1: PTC Therapeutics, Inc. - Revenue by Geography (%), 2016
Figure 13-2: Santhera Pharmaceuticals - Revenue by Business Segments (%), 2015
Figure 13-3: Santhera Pharmaceuticals - Revenue by Geography (%), 2015
Figure 13-4: Bristol Myers Squibb - Revenue by Business Segments (%), 2015
Figure 13-5: Bristol Myers Squibb - Revenue by Geography (%), 2015
Figure 13-6: FibroGen, Inc. - Revenue by Geography (%), 2016
Figure 13-7: Pfizer Inc. - Revenue by Business Segments (%), 2016
Figure 13-8: Pfizer Inc. - Revenue by Geography (%), 2016 



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