듀센형 근이영양증(DMD) : 신규 요법, 미충족 수요 및 TPP 인사이트 보고서(2026년)

Duchenne Muscular Dystrophy (DMD) Emerging Therapy and TPP Insights

Thelansis's "Duchenne Muscular Dystrophy (DMD) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Duchenne Muscular Dystrophy (DMD) Overview

Duchenne muscular dystrophy (DMD) is a severe, inherited neuromuscular disorder in which a missing structural protein leads to progressive muscle degeneration, historically managed through corticosteroids, supportive care, and, for a genetically defined subset of patients, exon-skipping therapies designed to partially restore that missing protein. The field's most consequential recent development is a one-time gene therapy delivering a shortened but functional version of the missing protein to muscle cells, a novel mechanism that reached the market on an accelerated pathway despite mixed trial results. That approval has proven contentious: reports of fatal liver toxicity in treated patients undercut early enthusiasm, even as longer-term follow-up data released more recently have renewed confidence by showing a sustained slowing of disease progression relative to how DMD typically unfolds untreated. Competitors are now advancing their own next-generation gene therapies, aiming to broaden eligibility and improve on this first mover's safety profile. For stakeholders, DMD captures both the promise and the real risk tolerance gene therapy demands of regulators and families.

Key Highlights

• In Germany, prevalent DMD cases are projected to increase from 2,171 in 2025 to 2,193 by 2035.
• DMD is a rare, progressive neuromuscular disorder caused by dystrophin gene mutations, leading to irreversible muscle degeneration.
• Improved supportive care has increased survival, resulting in a larger adult DMD population.
• Gene therapies and mutation-targeted treatments are transforming the treatment landscape.

Market Overview

• The Germany DMD market is projected to grow from $294M in 2025 to $819M by 2035.
• Market growth is driven by:
• Uptake of high-cost gene and exon-skipping therapies
• Expanded treatment eligibility and improved diagnosis
• Future market value will be heavily influenced by next-generation genetic therapies.

Insights driven by surveys with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs
• Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
• Pipeline analysis
• Target patients for emerging therapies
• Key companies
• Key mechanism of actions
• Launch date estimates, etc.
• Clinical trial landscape analysis
• Target patient segments
• Trial endpoints
• Trial design
• Recruitment criteria, etc.
• Unmet Needs and Opportunities
• Performance of key current therapies
• Top areas of unmet needs
• Opportunity sizing for key unmet needs
• Target Product Profiles
• Attributes and levels
• Physician likelihood of prescribing
• Expected patient shares
• KOL insights on key emerging therapies
• Level of awareness
• Expected use / line of therapy
• Extent to fulfil key unmet needs
• KOL quotes

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Solid Biosciences Inc.
• Sarepta Therapeutics, Inc.
• REGENXBIO Inc.
• Edgewise Therapeutics, Inc.
• Santhera Pharmaceuticals
• Daiichi Sankyo Co., Ltd.
• Pfizer
• Hoffmann-La Roche
• Insmed Gene Therapy LLC
• BioMarin Pharmaceutical
• Taiho Pharmaceutical Co., Ltd.
• Capricor Inc.
• NS Pharma, Inc.
• PTC Therapeutics
• Entrada Therapeutics, Inc.
• Satellos Bioscience, Inc.
• Dyne Therapeutics
• Nippon Shinyaku Co., Ltd.
• Avidity Biosciences, Inc.
• Cumberland Pharmaceuticals
• Precision BioSciences, Inc.
• Belief BioMed (Beijing) Co., Ltd.

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risk, etc.

2. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

• Key takeaways
• Scientific attributes
• Commercial attributes
• Product positioning

4. Primary Market Research

• Current treatment landscape
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Current challenges
• Unmet needs
• Emerging therapies
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

6. Regulatory and Reimbursement Environments (by country and payer insights)

7. Appendix (e.g., bibliography, methodology)