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Duchenne Muscular Dystrophy (DMD) Pipeline Insight Report 2025, Featuring Analysis of Vamorolone (Santhera), Givinostat (Italfarmaco), and Pamrevlumab (Fibrogen)

1. 75+ companies are developing drugs for Duchenne Muscular Dystrophy. 2. FibroGen's Pamrevlumab is advancing to Phase 3 for DMD. 3. Emerging therapies aim to improve quality of life for DMD patients. 4. There's significant competition among companies for DMD treatments. 5. Clinical trials for DMD are extensive, with various stages of drug development.

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FAQ

Why Bullish?

With Pamrevlumab progressing, FibroGen may capitalize on a significant market opportunity.

How important is it?

The progress of FibroGen's Pamrevlumab in DMD showcases its pipeline strength and market potential.

Why Long Term?

The potential for Pamrevlumab in DMD could lead to sustained revenue growth over time.

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Dublin, May 08, 2025 (GLOBE NEWSWIRE) -- The "Duchenne Muscular Dystrophy - Pipeline Insight, 2025" clinical trials has been added to ResearchAndMarkets.com's offering.This report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in Duchenne Muscular Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.Report HighlightsCompanies and academics are working to assess challenges and seek opportunities that could influence R&D Duchenne Muscular Dystrophy. The therapies under development are focused on novel approaches to treat/improve Duchenne Muscular Dystrophy.Duchenne Muscular Dystrophy Emerging Drugs Vamorolone: SantheraVamorolone 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.Givinostat: ItalfarmacoGivinostat, 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: FibrogenPamrevlumab 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).Duchenne Muscular Dystrophy: Therapeutic AssessmentThis segment of the report provides insights about the Duchenne Muscular Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:Major Players in Duchenne Muscular DystrophyThere 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 RochePhases The 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 candidatesDiscontinued & Inactive candidates Route of AdministrationDuchenne 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 OralIntravenousSubcutaneous Molecule Type Products have been categorized under various Molecule types such as Small moleculeCell TherapyPeptidesPolymerSmall moleculeGene therapy Product TypeDrugs have been categorized under various product types like Mono, Combination and Mono/Combination.Pipeline ActivitiesThe report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Duchenne Muscular Dystrophy therapeutic drugs key players involved in developing key drugs.Development ActivitiesThe report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Duchenne Muscular Dystrophy drugs. Key Questions Current Treatment Scenario and Emerging Therapies: How many companies are developing Duchenne Muscular Dystrophy drugs?How many Duchenne Muscular Dystrophy drugs are developed by each company?How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Duchenne Muscular Dystrophy?What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy therapeutics?What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?What are the clinical studies going on for Duchenne Muscular Dystrophy and their status?What are the key designations that have been granted to the emerging drugs? Key Players Santhera PharmaceuticalsSarepta TherapeuticsItalfarmacoWave Life Sciences Ltd.FibroGenEDG 5506 Edgewise TherapeuticsFordadistrogene movaparvovecDaiichi SankyoSarepta Therapeutics, Inc.ENCellTaiho PharmaceuticalSolid BiosciencesCapricorNippon ShinyakuHansa Biopharma Key Products VamoroloneDelandistrogene moxeparvovecGivinostatWVE N531PamrevlumabEDG 5506Fordadistrogene movaparvovecRenadirsenSRP 5051EN 001TAS-205SGT 001CAP 1002NS 089/NCNP 02UX 810 For more information about this clinical trials report visit https://www.researchandmarkets.com/r/7x02f9 About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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