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.
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Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
- June 2024:- Edgewise Therapeutics Inc.- A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy.
- June 2024:- Italfarmaco- Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy. This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to
- June 2024:- Hoffmann-La Roche- A Phase II Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Pediatric Patients With Duchenne Muscular Dystrophy (SHIELD DMD). 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.
- June 2024:- Pfizer- The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
- June 2024:- BioMarin Pharmaceutical- A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy. The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- June 2024:- Dyne Therapeutics- A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping. 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.
- 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.
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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).
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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
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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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