UK X-Linked Myotubular Myopathy (XLMTM) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2592

  • Format: PPT, PDF

UK X-linked Myotubular Myopathy (XLMTM) Therapeutics Market: Segmented by Diagnosis, Therapeutics, and Distribution Channels – Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

SKU: 2592 Categories: ,

Report Overview

The UK X-Linked Myotubular Myopathy (XLMTM) therapeutics market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

The X-linked myotubular myopathy (XLMTM) is the most common form of centronuclear myopathy (CNM). The XLMTM is a rare genetic neuromuscular disorder that is characterized by muscle weakness that is most typically severe but can range from mild to profound. Symptoms are often present at birth, though may develop later in infancy or early childhood. Rarely, symptoms may not present until adolescence or adulthood. Common symptoms include mild to profound muscle weakness, diminished muscle tone (hypotonia or “floppiness”), feeding difficulties, and potentially severe breathing complications (respiratory distress). Feeding difficulties and respiratory distress develop because of the weakness of the muscles that are involved in swallowing and breathing.

The XLMTM is caused by mutations to the myotubularin (MTM1) gene. The disorder is inherited as an X-linked condition. The disorder predominantly affects males, but female carriers, while typically asymptomatic, can develop a range of symptoms. According to a study conducted in the UK, it is estimated that the incidence of 17 per million births for severe X-linked myotubular myopathy (XLMTM) has been published in a report.

Market Growth Drivers

The rise in incidence and prevalence of X-Linked Myotubular Myopathy (XLMTM) is likely to propel market growth. Moreover, huge investment in research & development by key players is anticipated to drive the market growth over the forecast period. Several players are investing in the development of products/therapies like Gene therapy that is being studied as an approach to therapy for XLMTM. In gene therapy, the defective gene present in a patient is replaced with a normal gene to enable the production of the active enzyme and prevent the development and progression of the disease.

Furthermore, the introduction of new products by key players is anticipated to drive the X-Linked Myotubular Myopathy (XLMTM) market. The emergence of new therapies for acute phase or prevention of X-Linked Myotubular Myopathy (XLMTM) creates lucrative opportunities for the market over the forecast period.

Market Restraints

However, rising treatment costs and lack of awareness among patients and physicians about X-Linked Myotubular Myopathy are among the major factors acting as restraints and will continue to pose a challenge to the X-Linked Myotubular Myopathy treatment market over the forecast period.


1. UK X-Linked Myotubular Myopathy Therapeutics Market Overview………..
A. Market Size
2. Market Growth Drivers & Restraints…………………………………………………….
Market Growth Drivers
A. Increasing Incidence and Prevalence of X-Linked Myotubular Myopathy
B. Huge Investments in Research & Development Activities
C. Emergence of Novel Therapies for X-Linked Myotubular Myopathy Treatment
Market Restraints
A. Rising Treatment Costs
B. Lack of Awareness
3. Major Types of X-Linked Myotubular Myopathy…………………………………………
A. Nemaline Myopathy
B. Central Core Myopathy
C. Congenital Myopathy
D. Minimulticore Myopathy
E. Centronuclear Myopathy
4. UK X-Linked Myotubular Myopathy Therapeutics Market Segmentation…………………………………
A. By Diagnosis
I. Molecular Genetic Testing
II. Surgical Removal
III. Muscle Biopsy
IV. Specialised Tests
V. Others
B. By Therapeutics
I. Physical Therapy
II. Occupational Therapy
III. Non-steroidal Anti-inflammatory Drugs (NSAIDs)
IV. Corticosteroids
V. Gene Therapy
VI. Protein Replacement Therapy
VII. Genetic Counselling
VIII. Others
C. By Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. UK X-Linked Myotubular Myopathy Major Products Market Share……….…
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………………………………………………………………
A. Audentes Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of XLMTM therapeutics market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

Based on the diagnosis, the XLMTM treatment market can be bifurcated into

  • Molecular genetic testing
  • Surgical removal
  • Muscle biopsy
  • Specialized tests
  • Others

The diagnosis is based upon the identification of additional characteristic symptoms (e.g., cryptorchidism, long fingers and toes, macrocephaly), a detailed family history, a thorough clinical evaluation, and a variety of specialized tests. The diagnosis of XLMTM is confirmed through molecular genetic testing, which can detect mutations in the MTM1 gene causative of the disorder. Molecular genetic testing can detect a mutation in approximately 60%-98% of affected individuals and is available on a clinical basis.

By Therapeutics:

On the basis of therapeutics, the XLMTM therapeutics market can be categorized into

  • Physical therapy
  • Occupational therapy
  • Non-steroidal anti-inflammatory drugs (NSAIDs)
  • Corticosteroids
  • Gene therapy
  • Protein replacement therapy
  • Genetic counseling
  • Others

The treatment of affected individuals usually requires intensive medical intervention. Some affected individuals will require prolonged, constant ventilation support. In gene therapy, the defective gene present in a patient is replaced with a normal gene to enable the production of the active enzyme and prevent the development and progression of the disease. Better market growth can be projected over the forecast period.

By Distribution Channel:

In terms of distribution channels, the XLMTM therapeutics market can be classified into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

An increasing number of people are being hospitalized for X-Linked Myotubular Myopathy. Therefore, hospital pharmacies are likely to be the largest distribution channel during the forecast period, as the drugs for X-Linked Myotubular Myopathy are being sold on a large scale through the hospital pharmacies.

Top Major Players

The X-Linked Myotubular Myopathy (XLMTM) therapeutics market is an emerging market, and the prominent players in the market are likely to adopt organic as well as inorganic strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major Key player operating in the UK market includes Audentes Therapeutics Inc.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

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