Duchenne Muscular Dystrophy Market

DelveInsight's "Duchenne Muscular Dystrophy - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Duchenne Muscular Dystrophy, historical and forecasted epidemiology as well as the Duchenne Muscular Dystrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Duchenne Muscular Dystrophy market report provides current treatment practices, emerging drugs, Duchenne Muscular Dystrophy market share of the individual therapies, current and forecasted Duchenne Muscular Dystrophy market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Duchenne Muscular Dystrophy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.


Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan


Study Period: 2017-2030


Duchenne Muscular Dystrophy Disease Understanding and Treatment Algorithm

The DelveInsight Duchenne Muscular Dystrophy market report gives a thorough understanding of the Duchenne Muscular Dystrophy by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis and treatment. 


Duchenne muscular dystrophy (DMD) 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 (DMD) 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 minimising 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 counsellors.


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 (DMD) 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 yoghurt) 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


Duchenne Muscular Dystrophy Epidemiology  


As per the DelveInsight analysis, the total diagnosed Duchenne Muscular Dystrophy prevalent population in the 7 MM was 27,685 in 2017. 


The Duchenne Muscular Dystrophy epidemiology division provide insights about historical and current Duchenne Muscular Dystrophy patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.  


Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted Duchenne Muscular Dystrophy epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2017 to 2030. 


Country Wise- Duchenne Muscular Dystrophy Epidemiology 

The epidemiology segment also provides the Duchenne Muscular Dystrophy epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. 


Duchenne Muscular Dystrophy Drug Chapters

The drug chapter segment of the Duchenne Muscular Dystrophy (DMD) report encloses the detailed analysis of Duchenne Muscular Dystrophy (DMD) marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Duchenne Muscular Dystrophy (DMD) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.


Duchenne Muscular Dystrophy (DMD) Marketed Drugs 


Vyondys 53 (Golodirsen): Sarepta Therapeutics 

Vyondys 53, developed by Sarepta Threrapeutics, is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53.


Vyondys 53 (golodirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. It is a clear to slightly opalescent, colorless liquid, which is supplied in single-dose vials containing 100 mg golodirsen (50 mg/mL). Vyondys 53 is formulated as an isotonic phosphate buffered saline solution with an osmolality of 260 to 320 mOSM and a pH of 7.5. Each milliliter of Vyondys 53 contains: 50 mg golodirsen; 0.2 mg potassium chloride; 0.2 mg potassium phosphate monobasic; 8 mg sodium chloride and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH.

Products detail in the report…


Emflaza: PTC Therapeutics

Deflazacort is a corticosteroid prodrug, whose active metabolite, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. Emflaza, a corticosteroid, works as an anti-inflammatory and immunosuppressant. It has been shown to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce the incidence of curvature of the spine (scoliosis) in people with DMD. Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.


Emflaza was discovered and developed by Marathon Pharmaceuticls in patients five years of age and older. However, in April 2017, PTC Therapeutics completed an acquisition of all rights to Emflaza in the United States. PTC received FDA’s approval for label expansion of Emflaza to include DMD patients 2–5 years of age in June 2019.

Products detail in the report…


Exondys 51: Sarepta Therapeutics 

Exondys 51 is a manufactured and commercialized product of Sarepta Therapeutics. Exondys 51 is an antisense oligonucleotide indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Exon skipping is a treatment approach for specific genetic mutations that can restore production of the dystrophin protein.


Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Eteplirsen contains 30 linked subunits. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. 

Products detail in the report…


Translarna: PTC Therapeutics 

Translarna is indicated for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged two years and older. A “readthrough” drug, Translarna is designed to act by changing the way muscle cells interpret genetic information, coaxing them to produce a needed muscle protein called dystrophin despite the presence of a nonsense mutation in the dystrophin gene. Translarna forces the cell to ignore this abnormal premature stop signal, enabling the production of the full-length, functional protein. Thus, Translarna works as a “protein restoration therapy”, which means that it aims to facilitate the production of a functional protein in patients who cannot produce it normally.


Ataluren should be administered orally every day in three doses. The first dose should be taken in the morning, the second at midday, and the third in the evening. Recommended dosing intervals are six hours between morning and midday doses, six hours between midday and evening doses, and 12 hours between the evening dose and the first dose on the next day. The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday, and 20 mg/kg body weight in the evening (for a total daily dose of 40 mg/kg body weight).

Products detail in the report…


Duchenne Muscular Dystrophy (DMD) Emerging Drugs 


Casimersen: Sarepta Therapeutics

Sarepta Therapeutics is developing Casimersen (SRP-4045), which is an exon-skipping therapy to treat Duchenne muscular dystrophy (DMD) patients whose disease is amenable to skipping exon 45. Exon skipping addresses the underlying cause of DMD by promoting the production of a shorter, but still functional, dystrophin protein to stabilize or slow disease progression.


Casimersen is designed to skip exon 45 in the pre-mRNA of the DMD gene in order to enable the remaining exons to come back into frame and be read into a protein. Exon skipping is a treatment strategy in which sections of genetic code are “skipped” (spliced out, or left out) during the protein manufacturing process, allowing cells to create shortened but partially functional dystrophin protein, the muscle protein missing in DMD. Exon skipping is not a cure for DMD, but potentially could lessen the severe muscle weakness and atrophy that is the hallmark of the disease.

Products detail in the report…


Viltolarsen: Nippon Shinyaku (NS Pharma)

Viltolarsen (NS-065/NCNP-01), is a novel exon skipping morpholino oligomer nucleic acid drug, which is being developed to treat DMD patients with mutations of the dystrophin gene that are amenable to exon 53 skipping. Exon skipping is a budding therapy that is being developed for patients with DMD. Specialized molecules are created to skip over the non-working part of the dystrophin gene and allow pieces of the puzzle to attach.

Products detail in the report…


Givinostat: Italfarmaco 

Italfarmaco is developing Givinostat (also known as ITF2357), an orally available small molecule that acts as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. ITF2357 inhibits class I and II histone deacetylases (HDAC) and has demonstrated a potent anti-proliferative and pro-apoptotic activity against several hematologic malignancies both in vitro and in vivo. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. By blocking HDAC enzymes, givinostat is expected to ‘switch on’ the follistatin gene, thereby increasing the amount of the follistatin protein in muscle cells. Follistatin is expected to increase muscle mass and prevent muscle degeneration by opposing the effects of myostatin, a protein that causes fat and fibrotic tissue to build up in the muscle preventing muscle growth and regeneration. This is expected to improve the symptoms of DMD. The therapeutic candidate induces apoptosis of multiple myeloma and acute myelogenous leukemia cells and this apoptosis takes place following induction of p21 and down-modulation of Bcl-2 and Mcl-1 proteins. 

Products detail in the report…


Edasalonexent: Catabasis Pharmaceuticals

Edasalonexent (also known as CAT-1004; Edasa) is a novel investigational drug being evaluated as a potential treatment of Duchenne Muscular Dystrophy (DMD) by Catabasis Pharmaceuticals. CAT-1004 is an orally administered small molecule. Edasalonexent is a conjugate of salicylic acid and docosahexaenoic acid (DHA) that targets to inhibit activated NF-κB further dropping muscle degeneration and increased muscle regeneration. Edasalonexent is conducting phase III trials for Duchenne Muscular Dystrophy (DMD).


The United States Food and Drug Administration (FDA) has granted orphan drug, fast track and rare pediatric disease designations to edasalonexent for the treatment of DMD. The European Commission (EC) has granted orphan medicinal product designation to edasalonexent for the treatment of DMD.

Products detail in the report…

 


Duchenne Muscular Dystrophy Market


Duchenne Muscular Dystrophy Market Outlook


"According to DelveInsight, Duchenne Muscular Dystrophy Market is Anticipated to Grow With a CAGR of 31.8% for the Study Period of 2017-2030."


The Duchenne Muscular Dystrophy market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Duchenne Muscular Dystrophy market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of 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 annual cost of therapy, inclusion and exclusion criteria's, 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 market data are presented with relevant tables and graphs to give a clear view of the market at first sight. 

According to DelveInsight, Duchenne Muscular Dystrophy market in 7MM is expected to change in the study period 2017-2030.


Key Findings

This section includes a glimpse of the Duchenne Muscular Dystrophy (DMD) 7MM market.

  • The United States accounts for the largest market size of Duchenne Muscular Dystrophy (DMD), in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan. 
  • The market size of Duchenne Muscular Dystrophy (DMD) in the seven major markets was USD 266.06 Million in 2017.


The United States Market Outlook

This section provides the total Duchenne Muscular Dystrophy market size and market size by therapies in the United States.


EU-5 Countries: Market Outlook

The total Duchenne Muscular Dystrophy market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom is provided in this section.


Japan Market Outlook 

The total Duchenne Muscular Dystrophy market size and market size by therapies in Japan is also mentioned. 


Duchenne Muscular Dystrophy Key Companies


Duchenne Muscular Dystrophy Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Duchenne Muscular Dystrophy market or expected to get launched in the market during the study period 2017-2030. The analysis covers Duchenne Muscular Dystrophy market uptake by drugs; patient uptake by therapies; and sales of each drug.    

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.


Duchenne Muscular Dystrophy Pipeline Development Activities 

The report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Duchenne Muscular Dystrophy key players involved in developing targeted therapeutics. 


Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing, patent details and other information for Duchenne Muscular Dystrophy emerging therapies.


Reimbursement Scenario in Duchenne Muscular Dystrophy

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy. 


KOL- Views 

To keep up with current market trends, we take KOLs and SME's opinion working in Duchenne Muscular Dystrophy domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Duchenne Muscular Dystrophy market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs. 


Competitive Intelligence Analysis 

We perform Competitive and Market Intelligence analysis of the Duchenne Muscular Dystrophy Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.


Scope of the Report

  • The report covers the descriptive overview of Duchenne Muscular Dystrophy, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
  • Comprehensive insight has been provided into the Duchenne Muscular Dystrophy epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for Duchenne Muscular Dystrophy are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of Duchenne Muscular Dystrophy market; historical and forecasted is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Duchenne Muscular Dystrophy market


Report Highlights

  • In the coming years, Duchenne Muscular Dystrophy market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence Duchenne Muscular Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition       
  • Major players are involved in developing therapies for Duchenne Muscular Dystrophy. Launch of emerging therapies will significantly impact the Duchenne Muscular Dystrophy market
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Duchenne Muscular Dystrophy
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities


Duchenne Muscular Dystrophy Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Duchenne Muscular Dystrophy Pipeline Analysis
  • Duchenne Muscular Dystrophy Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies


Duchenne Muscular Dystrophy Report Key Strengths

  • 11 Years Forecast
  • 7MM Coverage 
  • Duchenne Muscular Dystrophy Epidemiology Segmentation
  • Key Cross Competition 
  • Highly Analyzed Market
  • Drugs Uptake


Duchenne Muscular Dystrophy Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers


Key Questions

Market Insights:

  • What was the Duchenne Muscular Dystrophy market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the Duchenne Muscular Dystrophy total market size as well as market size by therapies across the 7MM during the forecast period (2017-2030)?
  • What are the key findings pertaining to the market across 7MM and which country will have the largest Duchenne Muscular Dystrophy market size during the forecast period (2017-2030)?
  • At what CAGR, the Duchenne Muscular Dystrophy market is expected to grow in 7MM during the forecast period (2017-2030)?
  • What would be the Duchenne Muscular Dystrophy market outlook across the 7MM during the forecast period (2017-2030)?
  • What would be the Duchenne Muscular Dystrophy market growth till 2030, and what will be the resultant market Size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of the Duchenne Muscular Dystrophy?
  • What is the historical Duchenne Muscular Dystrophy patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of Duchenne Muscular Dystrophy in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Duchenne Muscular Dystrophy?
  • Out of all 7MM countries, which country would have the highest prevalent population of Duchenne Muscular Dystrophy during the forecast period (2017-2030)?
  • At what CAGR the population is expected to grow in 7MM during the forecast period (2017-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

  • What are the current options for the Duchenne Muscular Dystrophy treatment, along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Duchenne Muscular Dystrophy in the USA, Europe, and Japan?
  • What are the Duchenne Muscular Dystrophy marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of Duchenne Muscular Dystrophy?
  • How many therapies are developed by each company for Duchenne Muscular Dystrophy treatment?
  • How many are emerging therapies in mid-stage, and late stage of development for Duchenne Muscular Dystrophy treatment?
  • What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy therapies? 
  • What are the recent novel therapies, targets, mechanisms of action and 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 for the emerging therapies for Duchenne Muscular Dystrophy?
  • What are the global historical and forecasted market of Duchenne Muscular Dystrophy?


Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Duchenne Muscular Dystrophy market
  • To understand the future market competition in the Duchenne Muscular Dystrophy market and Insightful review of the key market drivers and barriers
  • Organize sales and marketing efforts by identifying the best opportunities for Duchenne Muscular Dystrophy in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors
  • Organize sales and marketing efforts by identifying the best opportunities for Duchenne Muscular Dystrophy market
  • To understand the future market competition in the Duchenne Muscular Dystrophy market


What is Duchenne Muscular Dystrophy (DMD)?

Duchenne Muscular Dystrophy (DMD) is a progressive form of muscular dystrophy that occurs primarily in males, though in rare cases may affect females too, causing progressive weakness and loss (atrophy) of skeletal and heart muscles.


What was the Duchenne Muscular Dystrophy (DMD) market size in the major markets?

The market size of DMD in the 7 MM was found to be USD 1,011 Million in 2017, during the study period (2017-2030).


Which geography accounted for the largest Duchenne Muscular Dystrophy (DMD) market size?

The United States accounts for the largest Duchenne Muscular Dystrophy (DMD) market size in comparison to EU5 and Japan.


What are the emerging drugs for Duchenne Muscular Dystrophy (DMD)?

Emerging therapies expected to impact Duchenne Muscular Dystrophy treatment market include Casimersen, Idebenone, Edasalonexent, Talditercept alfa, Givinostat, Arbekacin, PF-06939926, Ifetroban and many others.


Which are the leading companies in Duchenne Muscular Dystrophy (DMD) market?

Companies, like Sarepta Therapeutics (Casimersen [SRP-4045]), Santhera Pharmaceuticals (Idebenone), Catabasis Pharmaceuticals(Edasalonexent), Hoffmann-La Roche (Talditercept alfa), Italfarmaco (Givinostat), Nobelpharma (Arbekacin), Pfizer (PF-06939926), Cumberland Pharma (Ifetroban) and many others are working toward the development of new treatment therapies for Duchenne Muscular Dystrophy.


How is epidemiology segmented for Duchenne Muscular Dystrophy (DMD)?

Duchenne Muscular Dystrophy Epidemiology is segmented into total prevalent cases, total diagnosed prevalent population, age-specific diagnosed prevalence, mutation-specific diagnosed prevalence and prevalent population of associated comorbidities with Duchenne Muscular Dystrophy.

1 Key Insights

2 Duchenne Muscular Dystrophy (DMD) Market Overview at a Glance

2.1 Market Share (%) Distribution of Duchenne Muscular Dystrophy (DMD) in 2017

2.2 Market Share (%) Distribution of Duchenne Muscular Dystrophy (DMD) in 2030

3 Disease Background and Overview: Duchenne Muscular Dystrophy (DMD)

3.1 Introduction

3.2 Clinical Manifestations

3.3 Causes

3.3.1 Occurrence of Duchenne in Family

3.4 Inheritance of DMD

3.4.1 Females and DMD

3.5 Pathophysiology

3.6 Biomarkers

3.6.1 Tissue Composition

3.6.2 Genetic Makers

3.6.3 Proteins

3.6.4 Lipids and Metabolites

3.6.5 MicroRNA

3.7 Risk Factors

3.8 Diagnosis

3.9 Disease Progression

4 Recognized Establishments

4.1 United States

4.2 Europe

4.3 Japan

5 Epidemiology and Patient Population

5.1 Key Findings

5.1 7MM Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD)

6 Country Wise-Epidemiology of Duchenne Muscular Dystrophy (DMD)

6.1 United States

6.1.1 Assumptions and Rationale

6.1.2 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the United States

6.1.3 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United States

6.1.4 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United States

6.1.5 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in the United States

6.2 EU5 Countries

6.2.1 Assumptions and Rationale

6.3 Germany

6.3.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Germany

6.3.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Germany

6.3.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Germany

6.3.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Germany

6.4 France

6.4.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in France

6.4.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in France

6.4.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in France

6.4.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in France

6.5 Italy

6.5.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Italy

6.5.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Italy

6.5.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Italy

6.5.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Italy

6.6 Spain

6.6.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Spain

6.6.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Spain

6.6.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Spain

6.6.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Spain

6.7 United Kingdom

6.7.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the United Kingdom

6.7.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United Kingdom

6.7.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United Kingdom

6.7.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in the United Kingdom

6.8 Japan

6.8.1 Assumptions and Rationale

6.8.2 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Japan

6.8.3 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Japan

6.8.4 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Japan

6.8.5 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Japan

7 Treatment

7.1 Standard of care for DMD

7.1.1 Corticosteroids

7.1.2 Surgery

7.2 Therapeutic Strategies

7.2.1 Genetic Therapies

7.2.2 Cell therapy using muscle precursor cells or stem cells

7.2.3 Membrane stabilization and upregulation of cytoskeletal proteins

7.2.4 Treatment of Secondary Cascades

7.3 Medical Management

7.3.1 Braces, standing frames and wheelchairs

7.3.2 Cardiac Care

7.3.3 Contractures

7.3.4 Diet

7.3.5 Exercise

7.3.6 Physical and occupational therapy

7.3.7 Respiratory Care

7.3.8 Spinal Curvatures

7.4 Supportive treatment for DMD

7.5 Psychosocial management

8 General Guidelines on Duchenne Muscular Dystrophy (DMD)

9 United States Guidelines on Duchenne Muscular Dystrophy (DMD)

9.1 Efficacy of corticosteroids with regard to Duchenne muscular dystrophy (DMD) progression

9.1.1 Prednisone

9.1.2 Deflazacort

9.2 Optimal dosing regimen

9.2.1 Prednisone

10 European Guidelines on Duchenne Muscular Dystrophy (DMD)

11 Unmet Needs

12 Marketed Drugs

12.1 Vyondys 53 (Golodirsen): Sarepta Therapeutics

12.1.1 Drug Description

12.1.2 Regulatory Milestones

12.1.3 Other Development Activities

12.1.4 Safety and Efficacy

12.1.5 Product Profile

12.2 Emflaza: PTC Therapeutics

12.2.1 Drug Description

12.2.2 Regulatory Milestones

12.2.3 Other Development Activities

12.2.4 Safety and Efficacy

12.2.5 Product Profile

12.3 Exondys 51: Sarepta Therapeutics

12.3.1 Drug Description

12.3.2 Regulatory Milestones

12.3.3 Other Development Activities

12.3.4 Safety and Efficacy

12.3.5 Product Profile

12.4 Translarna: PTC Therapeutics

12.4.1 Drug Description

12.4.2 Regulatory Milestones

12.4.3 Other Development Activities

12.4.4 Safety and Efficacy

12.4.5 Product Profile

13 Emerging Drugs

13.1 Key Cross Competition

13.2 Casimersen: Sarepta Therapeutics

13.2.1 Product Description

13.2.2 Other Development Activities

13.2.3 Clinical Development

13.2.4 Safety and Efficacy

13.2.5 Product Profile

13.3 Puldysa (Idebenone): Santhera Pharmaceuticals

13.3.1 Product Description

13.3.2 Other Development Activities

13.3.3 Clinical Development

13.3.4 Safety and Efficacy

13.3.5 Product Profile

13.4 Givinostat: Italfarmaco

13.4.1 Product Description

13.4.2 Other Development Activities

13.4.3 Clinical Development

13.4.4 Safety and Efficacy

13.4.5 Product Profile

13.5 Edasalonexent: Catabasis Pharmaceuticals

13.5.1 Product Description

13.5.2 Other Development Activities

13.5.3 Clinical Development

13.5.4 Safety and Efficacy

13.5.5 Product Profile

13.6 Viltolarsen: Nippon Shinyaku (NS Pharma)

13.6.1 Product Description

13.6.2 Other Development Activities

13.6.3 Clinical Development

13.6.4 Safety and Efficacy

13.6.5 Product Profile

13.7 PF-06939926: Pfizer

13.7.1 Product Description

13.7.2 Other Development Activities

13.7.3 Clinical Development

13.7.4 Safety and Efficacy

13.7.5 Product Profile

13.8 Vamorolone: Santhera Pharmaceuticals/ReveraGen BioPharma

13.8.1 Product Description

13.8.2 Other Development Activities

13.8.3 Clinical Development

13.8.4 Safety and Efficacy

13.8.5 Product Profile

13.9 TAS-205: Taiho Pharmaceutical

13.9.1 Product Description

13.9.2 Other Development Activities

13.9.3 Clinical Development

13.9.4 Safety and Efficacy

13.9.5 Product Profile

13.10 Pamrevlumab (FG-3019): FibroGen

13.10.1 Product Description

13.10.2 Other Development Activities

13.10.3 Clinical Development

13.10.4 Safety and Efficacy

13.10.5 Product Profile

13.11 SRP-9001: Sarepta Therapeutics

13.11.1 Product Description

13.11.2 Other Development Activities

13.11.3 Clinical Development

13.11.4 Safety and Efficacy

13.11.5 Product Profile

13.12 Allogeneic Cardiosphere-Derived Cells (CAP-1002): Capricor

13.12.1 Product Description

13.12.2 Other Development Activities

13.12.3 Clinical Development

13.12.4 Safety and Efficacy

13.12.5 Product Profile

13.13 DS-5141b: Daiichi Sankyo

13.13.1 Product Description

13.13.2 Other Development Activities

13.13.3 Clinical Development

13.13.4 Safety and Efficacy

13.13.5 Product Profile

14 Duchenne Muscular Dystrophy (DMD): 7 Major Market Analysis

14.1 Key Findings

14.2 Market Size of Duchenne Muscular Dystrophy (DMD) in the 7MM

15 The United States Market Outlook

15.1 United States Market Size

15.1.1 Total Market size of Duchenne Muscular Dystrophy (DMD)

15.1.2 Market Size by Therapies

16 EU-5 Countries: Market Outlook

16.1 Germany

16.1.1 Total Market size of Duchenne Muscular Dystrophy (DMD) in Germany

16.1.2 Market Size by Therapies

16.2 France

16.2.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)

16.2.2 Market Size by Therapies

16.3 Italy

16.3.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)

16.3.2 Market Size by Therapies

16.4 Spain

16.4.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)

16.4.2 Market Size by Therapies

16.5 United Kingdom

16.5.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)

16.5.2 Market Size by Therapies

17 Japan: Market Outlook

17.1 Japan market Size

17.1.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)

17.1.2 Market Size by Therapies

18 Case Reports

18.1 Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents (United States)

19 Market Drivers

20 Market Barriers

21 SWOT Analysis for Duchenne Muscular Dystrophy

22 Appendix

22.1 Bibliography

22.2 Report Methodology

23 DelveInsight Capabilities

24 Disclaimer

25 About DelveInsight

Table 1 : 7MM Duchenne Muscular Dystrophy Epidemiology (2017-2030)

Table 2 : 7MM Duchenne Muscular Dystrophy Diagnosed and Treatable Cases (2017-2030)

Table 3 : Disease% Epidemiology in the United States (2017-2030)

Table 4 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in the United States (2017-2030)

Table 5 : Duchenne Muscular Dystrophy Epidemiology in Germany (2017-2030)

Table 6 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Germany (2017-2030)

Table 7 : Duchenne Muscular Dystrophy Epidemiology in France (2017-2030)

Table 8 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in France (2017-2030) 

Table 9 : Duchenne Muscular Dystrophy Epidemiology in Italy (2017-2030)

Table 10 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Italy (2017-2030)

Table 11 : Duchenne Muscular Dystrophy Epidemiology in Spain (2017-2030)

Table 12 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Spain (2017-2030)

Table 13 : Duchenne Muscular Dystrophy Epidemiology in the UK (2017-2030)

Table 14 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in the UK (2017-2030)

Table 15 : Duchenne Muscular Dystrophy Epidemiology in Japan (2017-2030)

Table 16 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Japan (2017-2030)

Table 17 : Drug Name, Clinical Trials by Recruitment status

Table 18 : Drug Name, Clinical Trials by Zone

Table 19 : Total Seven Major Market Size in USD, Million (2017-2030)

Table 20 : Region-wise Market Size in USD, Million (2017-2030)

Table 21 : 7MM-Market Size by Therapy in USD, Million (2017-2030)

Table 22 : United States Market Size in USD, Million (2017-2030)

Table 23 : United States Market Size by Therapy in USD, Million (2017-2030)

Table 24 : Germany Market Size in USD, Million (2017-2030)

Table 25 : Germany Market Size by Therapy in USD, Million (2017-2030)

Table 26 : France Market Size in USD, Million (2017-2030)

Table 27 : France Market Size by Therapy in USD, Million (2017-2030)

Table 28 : Italy Market Size in USD, Million (2017-2030)

Table 29 : Italy Market Size by Therapy in USD, Million (2017-2030)

Table 30 : Spain Market Size in USD, Million (2017-2030)

Table 31 : Spain Market Size by Therapy in USD, Million (2017-2030)

Table 32 : United Kingdom Market Size in USD, Million (2017-2030)

Table 33 : United Kingdom Market Size by Therapy in USD, Million (2017-2030)

Table 34 : Japan Market Size in USD, Million (2017-2030)

Table 35 : Japan Market Size by Therapy in USD, Million (2017-2030)

*The list of tables is not exhaustive; the final content may vary

Figure 1 : 7MM Duchenne Muscular Dystrophy Epidemiology (2017-2030)

Figure 2 : 7MM Duchenne Muscular Dystrophy Diagnosed and Treatable Cases (2017-2030)

Figure 3 : Duchenne Muscular Dystrophy Epidemiology in the United States (2017-2030)

Figure 4 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in the United States (2017-2030)

Figure 5 : Duchenne Muscular Dystrophy Epidemiology in Germany (2017-2030)

Figure 6 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Germany (2017-2030)

Figure 7 : Duchenne Muscular Dystrophy Epidemiology in France (2017-2030)

Figure 8 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in France (2017-2030) 

Figure 9 : Duchenne Muscular Dystrophy Epidemiology in Italy (2017-2030)

Figure 10 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Italy (2017-2030)

Figure 11 : Duchenne Muscular Dystrophy Epidemiology in Spain (2017-2030)

Figure 12 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Spain (2017-2030)

Figure 13 : Duchenne Muscular Dystrophy Epidemiology in the UK (2017-2030)

Figure 14 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in the UK (2017-2030)

Figure 15 : Duchenne Muscular Dystrophy Epidemiology in Japan (2017-2030)

Figure 16 : Duchenne Muscular Dystrophy Diagnosed and Treatable Cases in Japan (2017-2030)

Figure 17 : Drug Name, Clinical Trials by Recruitment status

Figure 18 : Drug Name, Clinical Trials by Zone

Figure 19 : Total Seven Major Market Size in USD, Million (2017-2030)

Figure 20 : Region-wise Market Size in USD, Million (2017-2030)

Figure 21 : 7MM-Market Size by Therapy in USD, Million (2017-2030)

Figure 22 : United States Market Size in USD, Million (2017-2030)

Figure 23 : United States Market Size by Therapy in USD, Million (2017-2030)

Figure 24 : Germany Market Size in USD, Million (2017-2030)

Figure 25 : Germany Market Size by Therapy in USD, Million (2017-2030)

Figure 26 : France Market Size in USD, Million (2017-2030)

Figure 27 : France Market Size by Therapy in USD, Million (2017-2030)

Figure 28 : Italy Market Size in USD, Million (2017-2030)

Figure 29 : Italy Market Size by Therapy in USD, Million (2017-2030)

Figure 30 : Spain Market Size in USD, Million (2017-2030)

Figure 31 : Spain Market Size by Therapy in USD, Million (2017-2030)

Figure 32 : United Kingdom Market Size in USD, Million (2017-2030)

Figure 33 : United Kingdom Market Size by Therapy in USD, Million (2017-2030)

Figure 34 : Japan Market Size in USD, Million (2017-2030)

Figure 35 : Japan Market Size by Therapy in USD, Million (2017-2030)

*The list of figures is not exhaustive; the final content may vary.

  • Sarepta Therapeutics
  • Italfarmaco
  • Catabasis Pharmaceuticals
  • Nippon Shinyaku (NS Pharma)
  • Pfizer
  • Santhera Pharmaceuticals
  • ReveraGen BioPharma
  • Taiho Pharmaceutical
  • FibroGen
  • Capricor
  • Daiichi Sankyo
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