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:
- Several key pharmaceutical companies, including Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Pfizer, Santhera Pharmaceuticals/ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Capricor, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, and others, are developing novel products to improve the Duchenne Muscular Dystrophy treatment outlook.
- The total Duchenne Muscular Dystrophy market size will include the market size of the potential upcoming therapies and current treatment regimens in the seven major markets.
- As per DelveInsight analysis, the Duchenne Muscular Dystrophy market is anticipated to witness growth at a considerable CAGR
Strategize your business goals by understanding market dynamics @ Duchenne Muscular Dystrophy Market Landscape
Duchenne Muscular Dystrophy Overview
Duchenne Muscular Dystrophy is the most common form of muscular dystrophy in childhood. It is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy.
DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3–5. The disease primarily affects boys, but in rare cases it can affect girls. DMD is inherited as an X-linked disease. X-linked genetic disorders are conditions caused by an abnormal gene on the X chromosome and manifest mostly in males. Females that have a defective gene present on one of their X chromosomes are carriers for that disorder. Carrier females usually do not display symptoms because females have two X chromosomes and only one carries the defective gene. Males have one X chromosome that is inherited from their mother and if a male inherits an X chromosome that contains a defective gene he will develop the disease.
The clinical hallmarks of DMD include weakness and wasting of various voluntary muscles of the body. In most advanced stages of the disease, the heart and gut muscles will be affected. Symptoms of DMD are usually noticed in boys between 1–6 years of age. A steady decline occurs in muscle strength between the ages of 6–11 years. By age 10, braces may be needed for walking. By age 13, most boys with DMD are using a wheelchair full-time. Children with DMD are often late walkers. In toddlers, parents may notice enlarged calf muscles. This enlargement is known as pseudohypertrophy, or ""false enlargement,"" because the muscle tissue is abnormal and may contain scar tissue. A preschooler with DMD may seem clumsy and fall often. Parents also may note that children have trouble climbing stairs, getting up from the floor or running.
Duchenne Muscular Dystrophy Diagnosis
The aim of care around diagnosis is to provide a correct and rapid diagnosis, allowing commencement of suitable interventions, enduring support and education, and minimizing the length and effect of a potentially protracted diagnostic process. Diagnosis should be done by a neuromuscular specialist who can assess the child clinically and can rapidly access and interpret appropriate investigations in the context of the clinical presentation. Family follow-up and support after diagnosis will often be augmented by support from geneticists and genetic counselors.
Suspicion of the diagnosis of DMD should be considered irrespective of family history and is usually triggered in one of three ways, namely, observation of abnormal muscle function in a male child, detection of an increase in serum creatine kinase tested for unrelated indications, after the discovery of increased transaminases (aspartate aminotransferase and alanine aminotransferase, which are produced by muscle as well as liver cells.
The diagnosis of DMD should thus be considered before liver biopsy in any male child with increased transaminases. Initial symptoms might include delayed walking, frequent falls, or diffi culty with running and climbing stairs. Although DMD is typically diagnosed at around 5 years of age, the diagnosis might be suspected much earlier because of delays in attainment of developmental milestones, such as independent walking or language; such delays have been documented prospectively by following patients with DMD identified by newborn screening.
Duchenne Muscular Dystrophy Treatment
Glucocorticoids, more precisely prednisone and deflazacort, are the main drug treatment for DMD. Corticosteroids have been used for over two decades and they are the only medication that has been shown to increase muscular strength. Ataluren is a newer medicine that has been developed to treat some children with DMD aged five or older who can still walk. Ataluren comes as granules provided in sachets. The contents of each sachet are mixed into liquids or semi-solid food (such as yogurt) and then swallowed.
The main therapeutic strategies for the treatment of DMD includes:
- Gene replacement or other genetic therapies linked to specific mutations to restore dystrophin production
- Membrane stabilization and/or upregulation of compensatory proteins
- Reduction of the inflammatory cascade and/or enhancement of muscle regeneration
Do you know the treatment paradigms for different countries? Download our Duchenne Muscular Dystrophy Market Sample Report
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:
- Duchenne Muscular Dystrophy Prevalence
- Age-Specific Duchenne Muscular Dystrophy Prevalence
- Gender-Specific Duchenne Muscular Dystrophy Prevalence
- Diagnosed and Treatable Cases of Duchenne Muscular Dystrophy
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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 Therapy Assessment
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
- Sarepta Therapeutics
- PTC Therapeutics
- Nippon Shinyaku
- Pfizer
- Santhera Pharmaceuticals/ReveraGen BioPharma
- Taiho Pharmaceutical
- FibroGen
- Capricor
- Daiichi Sankyo
- Italfarmaco
- Antisense Therapeutics
- Solid Biosciences
For more information, visit Duchenne Muscular Dystrophy Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Duchenne Muscular Dystrophy Market Report:
- 11 Years Forecast
- 7MM Coverage
- Descriptive overview of Duchenne Muscular Dystrophy, causes, signs and symptoms, diagnosis, treatment
- Comprehensive insight into Duchenne Muscular Dystrophy epidemiology in the 7MM
- Duchenne Muscular Dystrophy marketed and emerging therapies
- Duchenne Muscular Dystrophy companies
- Duchenne Muscular Dystrophy market drivers and barriers
Key Questions Answered in the Duchenne Muscular Dystrophy Market Report 2032:
- What was the Duchenne Muscular Dystrophy market share distribution in 2019, and how would it appear in 2032?
- What is the total Duchenne Muscular Dystrophy market size and the market size by therapy across the 7MM for the study period (2019-32)?
- What are the important findings from 7MM, and which country will have the greatest Duchenne Muscular Dystrophy market size from 2019-32?
- During the study period (2019-2032), at what CAGR is the Duchenne Muscular Dystrophy market projected to expand at 7MM?
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
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Duchenne Muscular Dystrophy Epidemiology 2032
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Duchenne Muscular Dystrophy Pipeline 2023
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