DelveInsight’s “Myotubular Myopathy Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Myotubular Myopathy market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Myotubular Myopathy drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Myotubular Myopathy treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Myotubular Myopathy: An Overview
Myotubular (Centronuclear) Myopathy represents a group of rare conditions characterized by the central location of the nucleus in muscle cells, which is normally found at the periphery. The term Myotubular myopathy is commonly used for the x-linked form.
The X-linked form of myotubular myopathy (XLMTM) is caused by a mutation in the MTM1 gene. MTM1 is needed to produce myotubularin, an enzyme thought to be involved in the development and maintenance of muscle cells. MTM1 gene mutations are considered to disrupt the normal role of myotubularin in muscle cell development and maintenance. This then causes muscle weakness and other signs and symptoms of X-linked Myotubular myopathy.
XLMTM is classified into severe (classic), moderate, and mild forms. Most affected individuals have the severe (classic) form of XLMTM. Moderate and mild forms of XLMTM are far less common. In the severe form, affected male infants exhibit extreme muscle weakness and hypotonia (floppiness) at or shortly after birth. Weakness of the muscles used to breathe and swallow can cause respiratory distress and feeding difficulties during infancy often noticeable within the first few days or weeks of life.
The treatment of XLMTM is directed toward the specific symptoms that are apparent in each individual. The treatment of affected individuals usually requires intensive medical intervention. Some affected individuals will need prolonged, constant ventilation support and the insertion of a feeding tube (gastrostomy). Physical and occupational therapy is recommended to improve muscle strength and prevent contractures.
Myotubular Myopathy Market Key Facts
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According to a study conducted by Petterson et al. (2008), in the UK, the incidence of X-linked myotubular myopathy was estimated to be 2/100,000 male births. The X-linked form usually gives rise to a severe phenotype in males, presenting usually at birth with a marked weakness and hypotonia, external ophthalmoplegia, and respiratory failure.
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According to a study by McEntagart et al., the median survival in the disease was 29 months. The median survival age varied by disease severity, with a 13-year survival of 22%, 80%, and 100% for severe, moderate, and mild phenotypes, respectively.
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According to the study by Servais et al. (2018), the pediatric point prevalence was estimated to be
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According to a study conducted by M. McEntegert et al. (2002), X-linked myotubular myopathy (XLMTM) is the most commonly recognized form of Myotubular myopathies affecting 1 in 50,000 newborn males worldwide.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Myotubular Myopathy pipeline therapies. It also thoroughly assesses the Myotubular Myopathy market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the market trend for each marketed Myotubular Myopathy drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Myotubular Myopathy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Myotubular Myopathy epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Myotubular Myopathy epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Myotubular Myopathy Epidemiology, Segmented as –
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Diagnosed Prevalent Cases of Myotubular Myopathy in the 7MM [2019–2032]
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Gender-specific Diagnosed Prevalent Cases of Myotubular Myopathy in the 7MM [2019–2032]
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Treatable Cases of Myotubular Myopathy in the 7MM [2019–2032]
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Severity-Specific Diagnosed Prevalent Cases of Myotubular Myopathy in the 7MM [2019–2032]
Myotubular Myopathy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Myotubular Myopathy market or expected to be launched during the study period. The analysis covers the Myotubular Myopathy market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Myotubular Myopathy drugs based on their sale and market share.
The report also covers the Myotubular Myopathy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Myotubular Myopathy companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Myotubular Myopathy Therapeutics Analysis
There is no known cure for myotubular myopathy (MTM), which is a rare genetic disorder that affects muscle development and function. However, there were some supportive treatments aimed at managing the symptoms and improving the quality of life for individuals with MTM. It is important for medical professionals who are familiar with myotubular myopathy to develop a personalized treatment plan based on the individual’s specific needs and the latest advancements in the field.
The treatment of affected individuals usually requires intensive medical intervention. Some affected individuals require prolonged, constant ventilation support. There are different methods for ventilation including noninvasive and invasive techniques. The decision about the duration of respiratory support is best made by the family in careful consultation with the patient’s physicians and other members of the healthcare team based on the specifics of their case.
Off-label use of drugs such as dantrolene, pyridostigmine and salbutamol appears to be of benefit in small open-label pilot studies, but those findings will have to be confirmed in larger double-blind, placebo-controlled clinical trials.
Research is ongoing to develop pharmacological interventions that could potentially improve muscle function and overall health in individuals with MTM. Companies all over the 7MM are persistently working towards the development of novel treatment therapies that can address the defects caused by MTM1 gene mutation.
Myotubular Myopathy Emerging Therapies and Key Companies
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Resamirigene bilparvovec (AT132): Astellas Pharma
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KT430: Astellas Pharma
And Many Others
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Myotubular Myopathy Competitive Intelligence Analysis
4. Myotubular Myopathy Market Overview at a Glance
5. Myotubular Myopathy Disease Background and Overview
6. Myotubular Myopathy Patient Journey
7. Myotubular Myopathy Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Myotubular Myopathy Treatment Algorithm, Current Treatment, and Medical Practices
9. Myotubular Myopathy Unmet Needs
10. Key Endpoints of Myotubular Myopathy Treatment
11. Myotubular Myopathy Marketed Therapies
12. Myotubular Myopathy Emerging Drugs and Latest Therapeutic Advances
13. Myotubular Myopathy Seven Major Market Analysis
14. Attribute Analysis
15. Myotubular Myopathy Market Outlook (In US, EU5, and Japan)
16. Myotubular Myopathy Companies Active in the Market
17. Myotubular Myopathy Access and Reimbursement Overview
18. KOL Views on the Myotubular Myopathy Market
19. Myotubular Myopathy Market Drivers
20. Myotubular Myopathy Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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