Press release
Duchenne Muscular Dystrophy (DMD) Pipeline Analysis, 2024 | Insights into Recent FDA, EMA, and PMDA Approvals, Evolving Treatment Dynamics, Clinical Trials, Emerging Therapies, and Key Companies
Duchenne Muscular Dystrophy Clinical Trial Updates 2024 | FDA Approvals and Focused Therapies: Vyondys 53 (Golodirsen), Emflaza, Exondys 51, Translarna, Amondys 45, Viltepso, PF-06939926, and Vamorolone.According to DelveInsight's analysis, over 75 key pharmaceutical and biotech companies are currently developing more than 75 pipeline drugs in the Duchenne Muscular Dystrophy (DMD) therapeutics landscape. These drugs vary in terms of Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Many of these therapies are in advanced stages of clinical development and are anticipated to launch in the coming years.
The report "Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024" by DelveInsight provides a comprehensive overview of current clinical and non-clinical development activities, as well as growth prospects within the DMD market.
This pipeline report includes a thorough commercial and clinical evaluation of pipeline products, spanning from the pre-clinical stage to market readiness. It offers detailed information on each drug, including its mechanism of action, clinical trial data, NDA approvals (if applicable), and product development activities, such as technology partnerships, mergers, acquisitions, funding, designations, and other relevant details.
To download our free sample report, visit: https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr
Recent developments in the duchenne muscular dystrophy pipeline segment:
In June 2024, REGENXBIO Inc. began enrolling a new cohort of boys aged 1 to 3 years for the Phase I/II AFFINITY DUCHENNE® trial. This study aims to assess the safety and efficacy of RGX-202 in treating Duchenne muscular dystrophy.
Understanding Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies and is the most common hereditary neuromuscular disease, affecting individuals regardless of race or ethnic background. The condition results from mutations in the dystrophin gene, leading to progressive degeneration and weakness of muscle fibers. Initially, patients may experience difficulty walking, but as the disease advances, they often lose the ability to perform daily activities and may require wheelchairs. Common complications include cardiac and orthopedic issues, with death typically occurring in the twenties due to respiratory muscle weakness or cardiomyopathy. Current treatments primarily involve glucocorticoids and physiotherapy to mitigate orthopedic complications.
Dystrophin is a large cytoskeletal protein crucial for linking the cytoskeleton to the cell membrane and extracellular matrix. It is found at the plasma membrane in both muscle and non-muscle tissues and is a key component of the dystrophin-glycoprotein complex (DGC), which serves as a structural unit in muscle. In DMD, the absence of dystrophin and DGC proteins results in increased membrane fragility and permeability, disrupted calcium homeostasis, and oxidative damage, all of which contribute to muscle cell necrosis. As individuals with DMD age, their muscles' regenerative capacity diminishes, leading to a gradual replacement of muscle fibers with connective and adipose tissue.
To know more in detail about duchenne muscular dystrophy pipeline segment, visit: https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr
Duchenne Muscular Dystrophy Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the emerging therapies for the treatment of Duchenne Muscular Dystrophy and the aggregate therapies developed by major pharma companies.
It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.
It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
The report evaluates the drugs that are 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.
It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Duchenne Muscular Dystrophy market.
The report is compiled using data and insights gathered from the researcher's proprietary databases, company and university websites, clinical trial registries, conference proceedings, SEC filings, investor presentations, and press releases from companies and universities, as well as other industry-specific third-party sources.
DelveInsight's 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 - The Report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROA, such as -
Oral
Intravenous
Subcutaneous
Molecule Type - Products have been categorized under various Molecule types, such as
Small molecule
Cell Therapy
Peptides
Polymer
Small molecule
Gene therapy
Duchenne Muscular Dystrophy Emerging Therapies - Mechanisms of Action (MOA) Types
Dipeptidyl peptidase I inhibitors
Peroxisome proliferator-activated receptor delta modulators
Cell replacements
Dystrophin expression stimulants
Connective tissue growth factor inhibitors
Dystrophin replacements
Immunostimulants
Gene modulators
Dystrophin expression modulators
Learn How the Ongoing Clinical & Commercial Activities will Affect the Duchenne Muscular Dystrophy Therapeutic Segment @
https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr
Duchenne Muscular Dystrophy Therapeutics Landscape
Approximately 75 key companies are working on therapies for Duchenne Muscular Dystrophy. Currently, Roche is at the forefront of the therapeutics market, with its drug candidates in the most advanced stages of clinical development compared to other major players in the field.
Leading Companies in the Duchenne Muscular Dystrophy Therapeutics Market Include:
Santhera Pharmaceuticals
Sarepta Therapeutics
Italfarmaco
Wave Life Sciences Ltd
FibroGen
Edgewise Therapeutics
Fordadistrogene movaparvovec
Daiichi Sankyo
Sarepta Therapeutics, Inc.
ENCell
Taiho Pharmaceutical
Solid Biosciences
Capricor
Nippon Shinyaku
Hansa Biopharma
And Many Others
Know more about leading players in the duchenne muscular dystrophy pipeline, visit: https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr
Duchenne Muscular Dystrophy Drugs Covered in the Report Include:
Vamorolone: Santhera
Givinostat: Italfarmaco
Pamrevlumab: Fibrogen
WVE N531: WaVe lifeSciences
CRISPR/Cas9 gene editing therapy: Vertex Pharmaceuticals
MA-0211: Mitobridge
BLS-M22: Bioleaders Corporation
CAP 1002: Capricor
DS-5141: Daiichi Sankyo
TAS-205: Taiho Pharmaceutical
And Many More
Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @ https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr
Table of Content
1. Report Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy Current Treatment Patterns
4. Duchenne Muscular Dystrophy - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Duchenne Muscular Dystrophy Late Stage Products (Phase-III)
7. Duchenne Muscular Dystrophy Mid-Stage Products (Phase-II)
8. Duchenne Muscular Dystrophy Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Duchenne Muscular Dystrophy Discontinued Products
13. Duchenne Muscular Dystrophy Product Profiles
14. Key Companies in the Duchenne Muscular Dystrophy Market
15. Key Products in the Duchenne Muscular Dystrophy Therapeutics Segment
16. Dormant and Discontinued Products
17. Duchenne Muscular Dystrophy Unmet Needs
18. Duchenne Muscular Dystrophy Future Perspectives
19. Duchenne Muscular Dystrophy Analyst Review
20. Appendix
21. Report Methodology
Contact Us:
Kritika Rehani
krehani@delveinsight.com
+14699457679
www.delveinsight.com
About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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