DelveInsight’s, "Duchenne Muscular Dystrophy Pipeline Insight 2024” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in Duchenne Muscular Dystrophy pipeline landscape. It covers the Duchenne Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Duchenne Muscular Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Explore our latest breakthroughs in Duchenne Muscular Dystrophy Research. Learn more about our innovative pipeline today! @ Duchenne Muscular Dystrophy Pipeline Outlook
Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
- In October 2024:- Hoffmann-La Roche- The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) age ≥ 8 to < 16 years old receiving corticosteroid therapy.
- In October 2024:- Sarepta Therapeutics Inc.- This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
- 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
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).
Learn more about Duchenne Muscular Dystrophy Drugs opportunities in our groundbreaking Duchenne Muscular Dystrophy Research and development projects @ Duchenne Muscular Dystrophy Unmet Needs
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
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
Table of Content
- 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
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