Press release
Duchenne Muscular Dystrophy Clinical Trials Analysis 2024: FDA Approval, Pipeline, Therapeutic Innovations, and Companies by DelveInsight | Vertex Pharma, Bioleaders Corporation, Wave Life Sciences, REGENXBIO, Daiichi Sankyo, Sarepta Therapeutics, more
Duchenne Muscular Dystrophy Clinical Trials
"Duchenne Muscular Dystrophy Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Duchenne Muscular Dystrophy Market.
The Duchenne Muscular Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
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Some of the key takeaways from the Duchenne Muscular Dystrophy Pipeline Report:
• Duchenne Muscular Dystrophy Companies across the globe are diligently working toward developing novel Duchenne Muscular Dystrophy treatment therapies with a considerable amount of success over the years.
• Duchenne Muscular Dystrophy companies working in the treatment market are Vertex Pharmaceuticals, Bioleaders Corporation, Wave Life Sciences Ltd., REGENXBIO Inc., Daiichi Sankyo, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Santhera, Italfarmaco, Roche/Sarepta Therapeutics, Edgewise Therapeutics, Sarepta Therapeutics, Wave Life Sciences Ltd, PepGen, Ultragenyx Pharmaceutical, and others, are developing therapies for the Duchenne Muscular Dystrophy treatment
• Emerging Duchenne Muscular Dystrophy therapies in the different phases of clinical trials are- CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, SRP-5051, TAS-205, Pamrevlumab, Vamorolone, Givinostat, Delandistrogene moxeparvovec, EDG 5506, SRP-5051, WVE N531, PGN EDO51, UX810, and others are expected to have a significant impact on the Duchenne Muscular Dystrophy market in the coming years.
• In May 2024, Pfizer has announced the death of a patient in the Phase II DAYLIGHT study of fordadistrogene movaparvovec, an experimental gene therapy for Duchenne muscular dystrophy (DMD). The trial aims to evaluate the safety and dystrophin expression in young boys with DMD.
• In May 2024, NS Pharma, a subsidiary of the Japanese pharmaceutical company Nippon Shinyaku, has reported initial results from the global Phase III RACER53 study of viltolarsen (NS-065 / NCNP-01) for treating Duchenne muscular dystrophy (DMD). The randomized, placebo-controlled study included 77 ambulatory male DMD patients and aimed to evaluate the safety and efficacy of an 80mg/kg weekly dose of viltolarsen compared to a placebo over 48 weeks. The primary endpoint, 'Time to Stand from Supine,' measured as velocity (rise/sec), showed a trend of increased velocity in the viltolarsen group from baseline after 48 weeks.
• In January 2024, ELEVIDYS (delandistrogene moxeparvovec) is a one-time gene therapy designed for ambulatory children aged four to five who have Duchenne muscular dystrophy (DMD), a disease marked by gradual muscle weakening. This treatment targets those with a confirmed mutation in the dystrophin gene, essential for muscle function. The therapy delivers a gene that produces a shortened version of dystrophin, called ELEVIDYS micro-dystrophin, to the muscle cells.
Duchenne Muscular Dystrophy Overview
The most prevalent genetic neuromuscular condition that affects people of all racial and ethnic backgrounds is Duchenne muscular dystrophy. Only men are affected; it affects 1/3,600 live births of infant boys. Some affected neonates may exhibit moderate hypotonia, but further symptoms are rarely noticeable at birth or throughout the first few months of life.
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Emerging Duchenne Muscular Dystrophy Drugs Under Different Phases of Clinical Development Include:
• Pamrevlumab: FibroGen
• Vamorolone: Santhera
• Givinostat: Italfarmaco
• Delandistrogene moxeparvovec: Roche/Sarepta Therapeutics
• CRISPR editing therapy: Vertex Pharmaceuticals
• BLS-M22: Bioleaders Corporation
• WVE-N531 :Wave Life Sciences Ltd.
• RGX-202: REGENXBIO Inc.
• DS-5141: Daiichi Sankyo
• SRP-5051: Sarepta Therapeutics
• TAS-205: Taiho Pharmaceutical
• EDG 5506: Edgewise Therapeutics
• SRP-5051: Sarepta Therapeutics
• WVE N531: Wave Life Sciences Ltd
• PGN EDO51: PepGen
• UX810: Ultragenyx Pharmaceutical
Duchenne Muscular Dystrophy Route of Administration
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
• Oral
• Intravenous
• Subcutaneous
Duchenne Muscular Dystrophy Molecule Type
Duchenne Muscular Dystrophy Products have been categorized under various Molecule types, such as
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy
Duchenne Muscular Dystrophy Pipeline Therapeutics Assessment
• Duchenne Muscular Dystrophy Assessment by Product Type
• Duchenne Muscular Dystrophy By Stage and Product Type
• Duchenne Muscular Dystrophy Assessment by Route of Administration
• Duchenne Muscular Dystrophy By Stage and Route of Administration
• Duchenne Muscular Dystrophy Assessment by Molecule Type
• Duchenne Muscular Dystrophy by Stage and Molecule Type
DelveInsight's Duchenne Muscular Dystrophy Report covers around 75+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration
Further Duchenne Muscular Dystrophy product details are provided in the report. Download the Duchenne Muscular Dystrophy pipeline report to learn more about the emerging Duchenne Muscular Dystrophy therapies
https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr
Some of the key companies in the Duchenne Muscular Dystrophy Therapeutics Market include:
Key companies developing therapies for Duchenne Muscular Dystrophy are - Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, and others.
Duchenne Muscular Dystrophy Pipeline Analysis:
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 key 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.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Download Sample PDF Report to know more about Duchenne Muscular Dystrophy drugs and therapies
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Duchenne Muscular Dystrophy Pipeline Market Drivers
• Robust Duchenne Muscular Dystrophy pipeline, development of Mutation Suppression therapies are some of the important factors that are fueling the Duchenne Muscular Dystrophy Market.
Duchenne Muscular Dystrophy Pipeline Market Barriers
• However, high cost of Duchenne Muscular Dystrophy treatments, regulatory procedure & and lack of standardized protocol and other factors are creating obstacles in the Duchenne Muscular Dystrophy Market growth.
Scope of Duchenne Muscular Dystrophy Pipeline Drug Insight
• Coverage: Global
• Key Duchenne Muscular Dystrophy Companies: Vertex Pharmaceuticals, Bioleaders Corporation, Wave Life Sciences Ltd., REGENXBIO Inc., Daiichi Sankyo, Sarepta Therapeutics, Taiho Pharmaceutical, FibroGen, Santhera, Italfarmaco, Roche/Sarepta Therapeutics, Edgewise Therapeutics, Sarepta Therapeutics, Wave Life Sciences Ltd, PepGen, Ultragenyx Pharmaceutical, and others
• Key Duchenne Muscular Dystrophy Therapies: CRISPR editing therapy, BLS-M22, WVE-N531, RGX-202, DS-5141, SRP-5051, TAS-205, Pamrevlumab, Vamorolone, Givinostat, Delandistrogene moxeparvovec, EDG 5506, SRP-5051, WVE N531, PGN EDO51, UX810, and others
• Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and Duchenne Muscular Dystrophy emerging therapies
• Duchenne Muscular Dystrophy Market Dynamics: Duchenne Muscular Dystrophy market drivers and Duchenne Muscular Dystrophy market barriers
Request for Sample PDF Report for Duchenne Muscular Dystrophy Pipeline Assessment and clinical trials
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Table of Contents
1. Duchenne Muscular Dystrophy Report Introduction
2. Duchenne Muscular Dystrophy Executive Summary
3. Duchenne Muscular Dystrophy Overview
4. Duchenne Muscular Dystrophy- Analytical Perspective In-depth Commercial Assessment
5. Duchenne Muscular Dystrophy Pipeline Therapeutics
6. Duchenne Muscular Dystrophy Late Stage Products (Phase II/III)
7. Duchenne Muscular Dystrophy Mid Stage Products (Phase II)
8. Duchenne Muscular Dystrophy Early Stage Products (Phase I)
9. Duchenne Muscular Dystrophy Preclinical Stage Products
10. Duchenne Muscular Dystrophy Therapeutics Assessment
11. Duchenne Muscular Dystrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Duchenne Muscular Dystrophy Key Companies
14. Duchenne Muscular Dystrophy Key Products
15. Duchenne Muscular Dystrophy Unmet Needs
16 . Duchenne Muscular Dystrophy Market Drivers and Barriers
17. Duchenne Muscular Dystrophy Future Perspectives and Conclusion
18. Duchenne Muscular Dystrophy Analyst Views
19. Appendix
20. About DelveInsight
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