Press release
Duchenne Muscular Dystrophy Pipeline Outlook Report 2025: Key 75+ Companies and Breakthrough Therapies Shaping the Future Landscape
Explore our latest breakthroughs in Duchenne Muscular Dystrophy Research. Learn more about our innovative pipeline today! @ Duchenne Muscular Dystrophy Pipeline Outlook [https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
* In August 2025, REGENXBIO Inc . announced a phase I/II/III study to evaluate the safety, tolerability, pharmacodynamics (microdystrophin protein levels), pharmacokinetic, and clinical efficacy of RGX-202 when administered IV as one-time dose to ambulant male participants with Duchenne.
* In August 2025, Pfizer conducted a study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
* DelveInsight's Duchenne Muscular Dystrophy pipeline report depicts a robust space with 75+ active players working to develop 75+ pipeline therapies for Duchenne Muscular Dystrophy treatment.
* The leading Duchenne Muscular Dystrophy Companies such as Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma , and others.
* Promising Duchenne Muscular Dystrophy Therapies such as Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02 , and others.
Stay informed about the cutting-edge advancements in Duchenne Muscular Dystrophy Treatments. Download for updates and be a part of the revolution in Musculoskeletal Care @ Duchenne Muscular Dystrophy Clinical Trials Assessment [https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Duchenne Muscular Dystrophy Emerging Drugs
* Vamorolone: Santhera
Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. This has the potential to 'dissociate' efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from patient quality of life. On September 2, 2020, Santhera exercised its option and obtained worldwide rights to vamorolone in Duchenne muscular dystrophy and all other indications. Santhera and ReveraGen expect to complete the rolling NDA submission to the U.S. FDA in June 2022.
* Givinostat: Italfarmaco
Givinostat, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed for the treatment of DMD and BMD. Since Givinostat acts on the pathogenetic events downstream of the genetic defects, it is potentially a treatment for the whole DMD and BMD population and to counter the disease pathogenetic events in all muscular districts.
* Pamrevlumab: Fibrogen
Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is advancing towards Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and pancreatic cancer and has been granted Orphan Drug Designation (ODD) in each of these indications, and is currently in a Phase 2 trial for Duchenne muscular dystrophy (DMD).
The Duchenne Muscular Dystrophy Pipeline Report Provides Insights into
* The report provides detailed insights about companies that are developing therapies for the treatment of Duchenne Muscular Dystrophy with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Duchenne Muscular Dystrophy Treatment.
* Duchenne Muscular Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Duchenne Muscular Dystrophy 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.
* 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.
Learn more about Duchenne Muscular Dystrophy Drugs opportunities in our groundbreaking Duchenne Muscular Dystrophy Research and development projects @ Duchenne Muscular Dystrophy Unmet Needs [https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Duchenne Muscular Dystrophy Companies
Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma , and others.
Duchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
* Oral
* Intravenous
* Subcutaneous
Duchenne Muscular Dystrophy Products have been categorized under various Molecule types such as
* Small molecule
* Cell Therapy
* Peptides
* Polymer
* Small molecule
* Gene therapy
Discover the latest advancements in Duchenne Muscular Dystrophy Treatment by visiting our website. Stay informed about how we're transforming the future of musculoskeletal @ Duchenne Muscular Dystrophy Market Drivers and Barriers, and Future Perspectives [https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Scope of the Duchenne Muscular Dystrophy Pipeline Report
* Coverage- Global
* Duchenne Muscular Dystrophy Companies- Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma , and others.
* Duchenne Muscular Dystrophy Therapies- Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02 , and others.
* Duchenne Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Duchenne Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
For a detailed overview of our latest research findings and future plans, read the full details of Duchenne Muscular Dystrophy Pipeline on our website @ Duchenne Muscular Dystrophy Drugs and Companies [https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
Table of Contents
* Introduction
* Executive Summary
* Duchenne Muscular Dystrophy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Duchenne Muscular Dystrophy- DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Delandistrogene moxeparvovec: Roche
* Drug profiles in the detailed report.....
* Mid-Stage Products (Phase II)
* SRP 5051: Sarepta Therapeutics
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I/II)
* WVE N531: Wave Life Sciences
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* EDG 5506: Edgewise Therapeutics
* Drug profiles in the detailed report.....
* Inactive Products
* Duchenne Muscular Dystrophy Key Companies
* Duchenne Muscular Dystrophy Key Products
* Duchenne Muscular Dystrophy- Unmet Needs
* Duchenne Muscular Dystrophy- Market Drivers and Barriers
* Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
* Duchenne Muscular Dystrophy Analyst Views
* Duchenne Muscular Dystrophy Key Companies
* Appendix
About Us
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
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