PUBLISHER: QYResearch | PRODUCT CODE: 1866606
PUBLISHER: QYResearch | PRODUCT CODE: 1866606
The global market for Duchenne Muscular Dystrophy (DMD) Therapeutics was estimated to be worth US$ 2170 million in 2024 and is forecast to a readjusted size of US$ 4776 million by 2031 with a CAGR of 13.1% during the forecast period 2025-2031.
Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal. The genetic change that causes Duchenne-a mutation in the DMD gene-happens before birth and can be inherited, or new mutations in the gene can occur spontaneously. Muscle weakness becomes increasingly noticeable between the ages of 3 and 5, and most patients use a wheelchair by the time they are 12. During adolescence, heart and breathing muscles weaken, leading to serious, life-threatening complications.
The Duchenne muscular dystrophy (DMD) treatment market is primarily driven by the following factors:
I. Disease Burden and Unmet Needs
High Disability and Mortality Rates:
DMD is a progressive muscle wasting disease. Patients typically lose the ability to walk around age 10 and die from cardiopulmonary failure between the ages of 20 and 30. With approximately 300,000 cases worldwide and over 70,000 in China, the disease poses a significant social burden.
If a female carrier gives birth to a boy, there is a 50% chance that the child will inherit the disease, creating a high family genetic risk.
Limitations of Existing Treatments:
Current mainstream treatments, including corticosteroids (such as deflazacort) and topical exon-skipping drugs (such as Exondys 51), can only delay the disease, not cure it, and require long-term use with significant side effects.
There is a lack of universal treatments, which only cover specific gene mutation types (such as exon 51/53 skipping), limiting their applicable population.
Improved Diagnosis Rate:
With improved medical infrastructure and urbanization, China's DMD diagnosis rate is expected to rise from approximately 30% in 2022 to over 50% in 2025, bringing more patients into the treatment-needing population.
II. R&D Progress and Pipeline Breakthroughs
Breakthroughs in Gene Therapy and Oligonucleotide Technologies:
Gene therapies, such as Sarepta's SRP-9001 (microdystrophin delivered via an AAVrh74 vector) and Faith Pharmaceuticals' BBM-D101 (engineered AAV vector), achieve long-term protein expression with a single dose. Clinical trials have shown a significant increase in the percentage of positive fibers.
Huida Gene's CRISPR-Cas12 gene editing therapy has entered clinical trials, targeting gene mutation repair.
Oligonucleotide therapies, including antisense oligonucleotides (ASOs) and next-generation peptide-coupled oligonucleotides (PPMOs), such as PepGen's PGN-EDO51 and Entrada's ENTR-601-44, enhance cellular uptake and lysosomal escape, improving efficacy and reducing dosage.
Active Domestic R&D Pipeline:
Over 10 DMD drugs have entered clinical trials in China, including gene editing (such as Huida Gene's CRISPR-Cas12), AAV gene therapy (Jinwei Bio's JWK007), and mini-protein drugs (Faith Pharmaceuticals' BBM-D101). Some have received FDA orphan drug designation and are expected to be launched after 2026.
III. Policy Support and Optimized Payment Environment
Policy Support for Rare Diseases:
US: The DUX Act extends the market exclusivity period for DMD orphan drugs to 7.5 years, and the accelerated approval process shortens drug launch time by 34%.
China: DMD will be included in the second round of rare disease listings in 2024, promoting domestic R&D. Medical insurance coverage has expanded, with vamorolone included in the national medical insurance negotiations. The price of domestically produced gene therapies is expected to drop below 2 million yuan by 2026.
Improved Affordability:
North America: Commercial insurance companies (such as UnitedHealthcare) have prioritized DMD gene therapy reimbursement, with medical insurance coverage exceeding 80%. In Europe, reimbursement rates for products that pass HTA assessment exceed 80%, but product launches lag 9-15 months behind those in the US.
Asia-Pacific: Markets such as China, Japan, and South Korea are experiencing significant growth, with policies and capital jointly driving therapeutic accessibility.
The DMD therapy market is driven by disease severity, technological breakthroughs, and favorable policies. Gene editing and RNA therapies will dominate the future, but high costs and production capacity bottlenecks (such as AAV vector production) still need to be overcome. Companies need to focus on technological innovation and payment model optimization to address unmet medical needs.
This report aims to provide a comprehensive presentation of the global market for Duchenne Muscular Dystrophy (DMD) Therapeutics, focusing on the total sales revenue, key companies market share and ranking, together with an analysis of Duchenne Muscular Dystrophy (DMD) Therapeutics by region & country, by Type, and by Application.
The Duchenne Muscular Dystrophy (DMD) Therapeutics market size, estimations, and forecasts are provided in terms of sales revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. With both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Duchenne Muscular Dystrophy (DMD) Therapeutics.
Market Segmentation
By Company
Segment by Type
Segment by Application
By Region
Chapter Outline
Chapter 1: Introduces the report scope of the report, global total market size. This chapter also provides the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.
Chapter 2: Detailed analysis of Duchenne Muscular Dystrophy (DMD) Therapeutics company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.
Chapter 3: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 4: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 5: Revenue of Duchenne Muscular Dystrophy (DMD) Therapeutics in regional level. It provides a quantitative analysis of the market size and development potential of each region and introduces the market development, future development prospects, market space, and market size of each country in the world.
Chapter 6: Revenue of Duchenne Muscular Dystrophy (DMD) Therapeutics in country level. It provides sigmate data by Type, and by Application for each country/region.
Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product revenue, gross margin, product introduction, recent development, etc.
Chapter 8: Analysis of industrial chain, including the upstream and downstream of the industry.
Chapter 9: Conclusion.