According to DelveInsight, over 75 leading companies are actively engaged in developing more than 75 treatment therapies for Duchenne Muscular Dystrophy, reflecting a strong and ongoing pipeline effort.
Duchenne Muscular Dystrophy Overview:
Duchenne muscular dystrophy (DMD) is a severe inherited neuromuscular disorder and the most prevalent type of muscular dystrophy worldwide, affecting individuals across all races and ethnicities. It is caused by mutations in the dystrophin gene, leading to progressive muscle weakness and degeneration. Symptoms typically begin with difficulty walking and progressively worsen, eventually resulting in loss of mobility and dependence on a wheelchair. Patients often experience cardiac and orthopedic complications, and most succumb to the disease in their twenties due to respiratory failure or cardiomyopathy. Current treatments, such as glucocorticoids and physiotherapy, aim to slow disease progression and manage symptoms.
Dystrophin is a vital cytoskeletal protein that connects the muscle cell structure to its surrounding matrix via the dystrophin-glycoprotein complex (DGC). This complex helps stabilize muscle fibers during contraction. In DMD, the absence of dystrophin and DGC proteins leads to fragile cell membranes, disrupted calcium balance, and increased oxidative stress, all of which contribute to muscle cell damage and death. Over time, as muscle regeneration declines, functional muscle tissue is replaced by connective and fat tissue, leading to further decline in muscle function.
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“Duchenne Muscular Dystrophy Pipeline Insight 2025” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Duchenne Muscular Dystrophy Therapeutics Market.
Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
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DelveInsight’s Duchenne Muscular Dystrophy (DMD) pipeline report highlights a strong and evolving landscape, with over 75 active companies working on more than 75 potential therapies for DMD treatment.
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In June 2024, REGENXBIO Inc. began enrolling boys aged 1 to 3 years in its Phase I/II AFFINITY DUCHENNE® trial, aimed at evaluating the safety and efficacy of RGX-202, a gene therapy for DMD.
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Leading companies such as Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences, FibroGen, Edgewise Therapeutics (EDG-5506), Daiichi Sankyo, ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, and Hansa Biopharma are actively advancing novel therapies to enhance the DMD treatment landscape.
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Promising candidates in the pipeline include Vamorolone, Givinostat, Pamrevlumab, among others, spanning various stages of clinical development.
Duchenne Muscular Dystrophy Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Duchenne Muscular Dystrophy Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Duchenne Muscular Dystrophy treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Duchenne Muscular Dystrophy market.
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Duchenne Muscular Dystrophy Emerging Drugs
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Vamorolone: Santhera
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Givinostat: Italfarmaco
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Pamrevlumab: Fibrogen
Duchenne Muscular Dystrophy Companies
More than 75 major companies are actively developing treatments for Duchenne Muscular Dystrophy. Among them, Roche has drug candidates that have progressed to the most advanced stage of development, Phase III clinical trials.
DelveInsight’s report covers around 75+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Duchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Duchenne Muscular Dystrophy Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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Duchenne Muscular Dystrophy Pipeline Therapeutic Assessment
• Duchenne Muscular Dystrophy Assessment by Product Type
• Duchenne Muscular Dystrophy By Stage
• Duchenne Muscular Dystrophy Assessment by Route of Administration
• Duchenne Muscular Dystrophy Assessment by Molecule Type
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Table of Content
1. Report Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy Current Treatment Patterns
4. Duchenne Muscular Dystrophy – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Duchenne Muscular Dystrophy Late-Stage Products (Phase-III)
7. Duchenne Muscular Dystrophy Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Duchenne Muscular Dystrophy Discontinued Products
13. Duchenne Muscular Dystrophy Product Profiles
14. Duchenne Muscular Dystrophy Key Companies
15. Duchenne Muscular Dystrophy Key Products
16. Dormant and Discontinued Products
17. Duchenne Muscular Dystrophy Unmet Needs
18. Duchenne Muscular Dystrophy Future Perspectives
19. Duchenne Muscular Dystrophy Analyst Review
20. Appendix
21. Report Methodology
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