Press release
Duchenne Muscular Dystrophy Pipeline, FDA Approvals, Clinical Trials Developments, and Companies 2024
Duchenne Muscular Dystrophy Pipeline
Key takeaways from the Duchenne Muscular Dystrophy Pipeline Report
• 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 include 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
• Promising Duchenne Muscular Dystrophy pipeline therapies include Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.
• October 2023: Sarepta Therapeutics Inc. announced a study of Phase 3 clinical trials for Delandistrogene Moxeparvovec. The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
• November 2023: Dyne Therapeutics announced a study of Phase 1 & 2 clinical trials for DYNE-251. The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
Request a sample and discover the recent breakthroughs happening in the Duchenne Muscular Dystrophy Pipeline landscape @ Duchenne Muscular Dystrophy Pipeline Outlook Report- https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Duchenne Muscular Dystrophy Overview
Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not exhibit a predilection for any race or ethnic group. Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness. This weakness may present initially with difficulty in ambulation but progressively advances to such an extent that affected patients are unable to carry out activities of daily living and must use wheelchairs.
For further information, refer to the detailed Duchenne Muscular Dystrophy Drugs Launch, Duchenne Muscular Dystrophy Developmental Activities, and Duchenne Muscular Dystrophy News, click here for Duchenne Muscular Dystrophy Ongoing Clinical Trial Analysis- https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Duchenne Muscular Dystrophy Emerging Drugs Profile
• Vamorolone: Santhera
• Givinostat: Italfarmaco
• Pamrevlumab: Fibrogen
Duchenne Muscular Dystrophy Pipeline Therapeutics Assessment
There are approx. 75+ key companies which are developing the therapies Duchenne Muscular Dystrophy. The companies which have their Duchenne Muscular Dystrophy drug candidates in the most advanced stage, i.e phase III include Roche
DelveInsight's Duchenne Muscular Dystrophy Pipeline 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) along with the details of
• Pre-clinical and Discovery stage candidates
• 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
• Oral
• Intravenous
• Subcutaneous
Duchenne Muscular Dystrophy Pipeline Products have been categorized under various Molecule types such as
• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy
Find out more about the Duchenne Muscular Dystrophy Pipeline Segmentation, Therapeutics Assessment, and Duchenne Muscular Dystrophy Emerging Drugs @ Duchenne Muscular Dystrophy Treatment Landscape- https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&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, 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 Therapies- Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.
• Duchenne Muscular Dystrophy Segmentation: Molecule Type, Mechanism of Action, Route of Administration, Product Type
Dive deep into rich insights for drugs for Duchenne Muscular Dystrophy Pipeline Companies and Therapies, click here @ Duchenne Muscular Dystrophy Unmet Needs and Analyst Views- https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Table of Content
1. Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Duchenne Muscular Dystrophy- DelveInsight's Analytical Perspective
7. Late Stage Products (Phase III)
8. Delandistrogene moxeparvovec: Roche
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. SRP 5051: Sarepta Therapeutics
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. WVE N531: Wave Life Sciences
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. EDG 5506: Edgewise Therapeutics
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Duchenne Muscular Dystrophy Key Companies
21. Duchenne Muscular Dystrophy Key Products
22. Duchenne Muscular Dystrophy- Unmet Needs
23. Duchenne Muscular Dystrophy- Market Drivers and Barriers
24. Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
25. Duchenne Muscular Dystrophy Analyst Views
26. Duchenne Muscular Dystrophy Key Companies
27. Appendix
Got Queries? Find out the related information on Duchenne Muscular Dystrophy Mergers and acquisitions, Duchenne Muscular Dystrophy Licensing Activities @ Duchenne Muscular Dystrophy Emerging Drugs, and Recent Trends- https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
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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.
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: ybhardwaj@delveinsight.com
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