Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033

Powered by

All the vital news, analysis, and commentary curated by our industry experts.

This report covers the 7MM (US, France, Germany, Italy, Spain, UK, Japan) and provides an Excel-based forecast model for the DMD market through 2033.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. Dystrophin is part of a group of proteins, which together form the dystrophin-associated protein complex (DPC). These proteins form a transmembrane scaffold that is crucial for the structure of the muscle tissue, protecting muscle fibers from injury during muscle contraction and relaxation. DMD is an X-linked recessive disorder mainly affecting males, with female carriers.

The DMD market across the 7MM was valued at $2.3 billion in 2023. Over the 10-year forecast period, the market is projected to grow at a CAGR of 8.5%, reaching $5.2 billion by 2033. The major driver for this growth will be the launch of several pipeline agents: Roche’s gene therapy, Capricor’s cell therapy deramiocel, PTC Therapeutics’ ataluren in the US, and other first-in-class therapies.

Scope

Overview of DMD, including epidemiology, symptoms, diagnosis, and disease management.

Annualized DMD treatment market revenue, cost of therapy per patient, and treatment usage patterns forecast from 2023 to 2033.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications of these factors for the DMD therapeutics market.

Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for DMD treatment. The most promising candidates in Phase III and Phase IIb development are profiled.

Analysis of the current and future market competition in the global DMD therapeutics market. Insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Pipeline analysis: Emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.

Key Highlights

Report deliverables include a PowerPoint report and Excel-based forecast model

Forecasts includes the 7MM

Forecasts covers from 2023-2033

Reasons to Buy

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

Develop business strategies by understanding the trends shaping and driving the 7MM DMD therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the 7MM DMD therapeutics market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Sarepta Therapeutics Inc
Nippon Shinyaku Co Ltd
NS Pharma Inc
PTC Therapeutics Ltd
Santhera Theraeputics
Catalyst Pharmaceuticals Inc
Italfarmaco SpA
Capricor Therapeutics Inc
Roche (F. Hoffmann-La Roche)
Taiho Pharmaceutical
Edgewise Therapeutics

Table of Contents

Table of Contents

1. Preface

1.1. Contents

1.2. Abbreviations

1.3. Related Reports

2. Executive Summary

3. Disease Overview

3.1. Overview of Duchenne Muscular Dystrophy

3.2. Classification of DMD

3.3. Etiology of DMD

3.4. Pathophysiology of DMD

3.5. Diagnostic Paradigm

3.6. DMD SWOT Analysis

4. Epidemiology

4.1. Diagnosed Incident Cases of DMD, Men, Ages 0–39 Years, 2023–33

4.2. Diagnosed Prevalent Cases of DMD, Men, Ages 0–39 Years, 2023–33

4.3. Age-Specific Trends in Diagnosed Prevalent Cases of DMD, Men, 2023

4.4. Diagnosed Prevalent Cases of DMD by Severity, Men, Ages 0–39 Years, 2023

4.5. Diagnosed Prevalent Cases of DMD with a Specific Mutation, Men, Ages 0–39 Years, 2023

4.6. Diagnosed Prevalent Cases of DMD with Large Mutation Subtypes, Men, Ages 0–39 Years, 2023

4.7. Diagnosed Prevalent Cases of DMD with Small Mutation Subtypes, Men, Ages 0–39 Years, 2023

4.8. Diagnosed Prevalent Cases of DMD by Applicability of Therapies, Men, Ages 0–39 Years, 2023

4.9. Sources Used to Forecast Diagnosed Incident Cases of DMD

4.10. Sources Used to Forecast Diagnosed Prevalent Cases of DMD

4.11. Sources Used to Forecast Diagnosed Prevalent Cases of DMD by Severity

4.12. Sources Used to Forecast Diagnosed Prevalent Cases of DMD with a Specific Mutation, Diagnosed Prevalent Cases of DMD with Large Mutation Subtypes, Diagnosed Prevalent Cases of DMD with Small Mutation Subtypes

4.13. Sources Used to Forecast Diagnosed Prevalent Cases of DMD by Applicability of Therapies

4.14. Sources and Methodology – Diagnosed Incident Cases and Diagnosed Prevalent Cases of DMD

4.15. Sources and Methodology – Diagnosed Prevalent Cases of DMD by Severity, Specific Mutation, and Applicability of Therapies

5. Current Treatment Options

5.1. Treatment Paradigm

5.2. Current Treatments for DMD

5.3. Product Profile: Exondys 51 (eteplirsen)

5.4. Product Profile: Amondys 45 (casimersen)

5.5. Product Profile: Vyondys 53 (golodirsen)

5.6. Product Profile: Viltepso (viltolarsen)

5.7. Product Profile: Exon-Skipping Therapies

5.8. Product Profile: Emflaza (deflazacort)

5.9. Product Profile: Agamree (vamorolone)

5.10. Product Profile: Prednisolone

5.11. Product Profile: Steroids

5.12. Product Profile: Duvyzat (givinostat hydrochloride)

5.13. Product Profile: Elevidys (delandistrogene moxeparvovec-rokl)

5.14. Product Profile: Gene Therapy

5.15. Product Profile: Translarna (ataluren)

5.16. Product Profile: Translarna

6. Unmet Needs and Opportunities

6.1. Unmet Needs in DMD

6.2. Delay or Reverse Disease Regression

6.3. Access to Treatments for Non-ambulatory Patients

6.4. Cost of Therapies

7. R&D Strategies

7.1. Trends in Clinical Trial Design in DMD (1/2)

7.2. Trends in Clinical Trial Design in DMD (2/2)

7.3. Trends in Deal-Making in DMD

7.4. Top 10 Deals for DMD

8. Pipeline Assessment

8.1. DMD Pipeline Overview

8.2. Late-Stage Pipeline Agents for DMD

8.3. Product Profile: NS Pharma’s Viltolarsen

8.4. Product Profile: NS Pharma’s Brogidirsen

8.5. Product Profile: Italfarmaco’s Givinostat

8.6. Product Profile: Roche/Sarepta Therapeutics’ delandistrogene moxeparvovec (SRP-9001)

8.7. Product Profile: Gene Therapy

8.8. Product Profile: PTC Therapeutics’ Translarna (ataluren)

8.9. Product Profile: Capricor’s Deramiocel

8.10. Product Profile: Taiho Pharmaceutical’s Pizuglanstat (TAS-205)

8.11. Product Profile: Edgewise Therapeutic’s Sevasemten

8.12. Clinical Trials in the DMD Space

9. Market Outlook

9.1. DMD Market Forecast (1/2)

9.2. DMD Market Forecast (2/2)

9.3. Global Market Drivers and Barriers

10. Appendix

10.1. Primary Research: KOL Information

10.2. Bibliography

10.3. About the Authors

11. Contact Us

Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033 standard reports
Currency USD
$17,995

Can be used by individual purchaser only

$53,985

Can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company


Undecided about purchasing this report?

Enquire Before Buying Download FREE Resources

Get in touch to find out about multi-purchase discounts

reportstore@globaldata.com
Tel +44 20 7947 2745

Sample Report

Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033 was curated by the best experts in the industry and we are confident about its unique quality. However, we want you to make the most beneficial decision for your business, so we offer free sample pages to help you:

  • Assess the relevance of the report
  • Evaluate the quality of the report
  • Justify the cost

Download your copy of the sample report and make an informed decision about whether the full report will provide you with the insights and information you need.

Below is a sample report to understand what you are buying

See what our customers are saying
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image

“The GlobalData platform is our go-to tool for intelligence services. GlobalData provides an easy way to access comprehensive intelligence data around multiple sectors, which essentially makes it a one-for-all intelligence platform, for tendering and approaching customers.

GlobalData is very customer orientated, with a high degree of personalised services, which benefits everyday use. The highly detailed project intelligence and forecast reports can be utilised across multiple departments and workflow scopes, from operational to strategic level, and often support strategic decisions. GlobalData Analytics and visualisation solutions has contributed positively when preparing management presentations and strategic papers.”

Business Intelligence & Marketing Manager, SAL Heavy Lift

“COVID-19 has caused significant interference to our business and the COVID-19 intelligence from GlobalData has helped us reach better decisions around strategy. These two highlights have helped enormously to understand the projections into the future concerning our business units, we also utilise the project database to source new projects for Liebherr-Werk to use as an additional source to pitch for new business.”

Market Analyst & Management, Liebherr-Werk

Your daily news has saved me a lot of time and keeps me up-to-date with what is happening in the market, I like that you almost always have a link to the source origin. We also use your market data in our Strategic Business Process to support our business decisions. By having everything in one place on the Intelligence Center it has saved me a lot of time versus looking on different sources, the alert function also helps with this.

Head of Key Accounts, Saab AB

Having used several other market research companies, I find that GlobalData manages to provide that ‘difficult-to-get’ market data that others can’t, as well as very diverse and complete consumer surveys.

Marketing Intelligence Manager, Portugal Foods

Our experience with GlobalData has been very good, from the platform itself to the people. I find that the analysts and the account team have a high level of customer focus and responsiveness and therefore I can always rely on. The platform is more holistic than other providers. It is convenient and almost like a one stop shop. The pricing suite is highly competitive and value for our organisation.

I like reports that inform new segments such as the analysis on generation Z, millennials, the impact of COVID 19 to our banking customers and their new channel habits. Secondly the specialist insight on affluent sector significantly increases our understanding about this group of customers. The combination of those give us depth and breadth of the evolving market.

I’m in the business of answering and helping people make decisions so with the intelligence center I can do that, effectively and efficiently. I can share quickly key insights that answer and satisfy our country stakeholders by giving them many quality studies and primary research about competitive landscape beyond the outlook of our bank. It helps me be seen as an advisory partner and that makes a big difference. A big benefit of our subscription is that no one holds the whole data and because it allows so many people, so many different parts of our organisation have access, it enables all teams to have the same level of knowledge and decision support.

Head of Customer Insight and Research, Standard Chartered

“I know that I can always rely on Globaldata’s work when I’m searching for the right consumer and market insights. I use Globaldata insights to understand the changing market & consumer landscape and help create better taste & wellbeing solutions for our customers in food, beverage and healthcare industries.

Globaldata has the right data and the reports are of very high quality compared to your competitors. Globaldata not only has overall market sizes & consumer insights on food & beverages but also provides insights at the ingredient & flavour level. That is key for B2B companies like Givaudan. This way we understand our customers’ business and also gain insight to our unique industry”

Head of Consumer Sensory Insights, Givaudan

GlobalData provides a great range of information and reports on various sectors that is highly relevant, timely, easy to access and utilise.  The reports and data dashboards help engagement with clients; they provide valuable industry and market insights that can enrich client conversations and can help in the shaping of value propositions. Moreover, using GlobalData products has helped increase my knowledge of the finance sector, the players within it, and the general threats and opportunities.

I find the consumer surveys that are carried out to be extremely beneficial and not something I have seen anywhere else. They provided an insightful view of why and which consumers take (or don’t) particular financial products. This can help shape conversations with clients to ensure they make the right strategic decisions for their business.

One of the challenges I have found is that data in the payments space is often piecemeal. With GD all of the data I need is in one place, but it also comes with additional market reports that provide useful extra context and information. Having the ability to set-up alerts on relevant movements in the industry, be it competitors or customers, and have them emailed directly to me, ensures I get early sight of industry activity and don’t have to search for news.

Senior Account Manager, TSYS
Go even deeper with GlobalData Intelligence Center

Every Company Report we produce is powered by the GlobalData Intelligence Center.

Subscribing to our intelligence platform means you can monitor developments at Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033 in real time.

  • Access a live Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033 dashboard for 12 months, with up-to-the-minute insights.
  • Fuel your decision making with real-time deal coverage and media activity.
  • Turn insights on financials, deals, products and pipelines into powerful agents of commercial advantage.