The Duchenne Muscular Dystrophy market growth is driven by factors like increase in the prevalence of Duchenne Muscular Dystrophy, investments in research and development, entry of emerging therapies during the study period 2019-2032.
The Duchenne Muscular Dystrophy market report also offers comprehensive insights into the Duchenne Muscular Dystrophy market size, share, Duchenne Muscular Dystrophy epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Duchenne Muscular Dystrophy market size growth forward.
Some of the key highlights from the Duchenne Muscular Dystrophy Market Insights Report:
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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. Cardiac and orthopedic complications are common, and death usually occurs in the twenties due to respiratory muscle weakness or cardiomyopathy. Current therapy is centered on treatment with glucocorticoids and physiotherapy to prevent orthopedic complications.
Dystrophin is a large cytoskeletal protein that facilitates interactions between the cytoskeleton, cell membrane, and extracellular matrix. It is located at the plasma membrane in both muscle and non-muscle tissues. Dystrophin is a critical part of the dystrophin-glycoprotein complex (DGC), which plays an important role as being a structural unit of muscle. In DMD, both dystrophin and DGC proteins are missing, leading to excessive membrane fragility and permeability, dysregulation of calcium homeostasis, oxidative damage. These factors play a crucial role in muscle cell necrosis. As patients with DMD age, the regenerative capacity of the muscles appears to be exhausted, and connective and adipose tissue gradually replaces muscle fibers.
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Duchenne Muscular Dystrophy Epidemiology Segmentation
DelveInsight’s Duchenne Muscular Dystrophy market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Duchenne Muscular Dystrophy historical patient pools and forecasted Duchenne Muscular Dystrophy patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Duchenne Muscular Dystrophy Market report proffers epidemiological analysis for the study period 2019-32 in the 7MM segmented into:
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Recent Breakthroughs in the Duchenne Muscular Dystrophy Market
1. FDA advisers narrowly back first gene therapy for muscular dystrophy
2. FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation
Duchenne Muscular Dystrophy Treatment Market
The Duchenne Muscular Dystrophy market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Duchenne Muscular Dystrophy market trends by analyzing the impact of current Duchenne Muscular Dystrophy therapies on the market, unmet needs, drivers and barriers, and demand for better technology.
This segment gives a thorough detail of Duchenne Muscular Dystrophy market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Duchenne Muscular Dystrophy market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the Duchenne Muscular Dystrophy market in 7MM is expected to witness a major change in the study period 2019-2032.
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).
Duchenne Muscular Dystrophy Key Companies
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Scope of the Duchenne Muscular Dystrophy Market Report:
Key Questions Answered in the Duchenne Muscular Dystrophy Market Report 2032:
Table of Contents:
1 Duchenne Muscular Dystrophy Market Key Comprehensive Insights
2 Duchenne Muscular Dystrophy Market Report Introduction
3 Competitive Intelligence Analysis for Duchenne Muscular Dystrophy
4 Duchenne Muscular Dystrophy Market Analysis Overview at a Glance
5 Executive Summary of Duchenne Muscular Dystrophy
6 Duchenne Muscular Dystrophy Epidemiology and Market Methodology
7 Duchenne Muscular Dystrophy Epidemiology and Patient Population
8 Duchenne Muscular Dystrophy Patient Journey
9 Duchenne Muscular Dystrophy Treatment Algorithm, Duchenne Muscular Dystrophy Current Treatment, and Medical Practices
10 Key Endpoints in Duchenne Muscular Dystrophy Clinical Trials
11 Duchenne Muscular Dystrophy Marketed Therapies
12 Duchenne Muscular Dystrophy Emerging Therapies
13 Duchenne Muscular Dystrophy: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Duchenne Muscular Dystrophy
16 Duchenne Muscular Dystrophy Market Key Opinion Leaders Reviews
18 Duchenne Muscular Dystrophy Market Drivers
19 Duchenne Muscular Dystrophy Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
Related Reports:
Duchenne Muscular Dystrophy Epidemiology 2032
DelveInsight’s “Duchenne Muscular Dystrophy – Epidemiology Forecast to 2032” report delivers an in-depth understanding of the disease, historical and forecasted Duchenne Muscular Dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Duchenne Muscular Dystrophy Pipeline 2023
“Duchenne Muscular Dystrophy Pipeline Insights, 2023” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Duchenne Muscular Dystrophy market. A detailed picture of the Duchenne Muscular Dystrophy pipeline landscape is provided, which includes the disease overview and Duchenne Muscular Dystrophy treatment guidelines.
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