DelveInsight’s “Metachromatic Leukodystrophy Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Metachromatic Leukodystrophy 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 Metachromatic Leukodystrophy drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Metachromatic Leukodystrophy 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.
Metachromatic Leukodystrophy: An Overview
According to the National Institute of Health (NIH), Metachromatic Leukodystrophy (MLD) is a rare inherited disorder characterized by the accumulation of fats called sulfatides in cells. This accumulation especially affects cells in the nervous system that produce myelin, the substance that insulates and protects nerves. Nerve cells covered by myelin make up a tissue called white matter. This condition is called metachromatic leukodystrophy because when viewed under a microscope, sulfatide accumulation in cells appears as granules that are colored differently than other cellular material (metachromatic).
MLD is caused due to mutations in the ARSA gene, which provides instructions for making the enzyme arylsulfatase A. This enzyme is located in cellular structures called lysosomes, which are the cell’s recycling centers. Within lysosomes, arylsulfatase A helps break down sulfatides. A few individuals with metachromatic leukodystrophy have mutations in the PSAP gene. This gene provides instructions for making a protein that is broken up (cleaved) into smaller proteins that assist enzymes in breaking down various fats. One of these smaller proteins is called saposin B; this protein works with arylsulfatase A to break down sulfatides.
Metachromatic Leukodystrophy Market Key Facts
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According to the National Organization for Rare Diseases (NORD), the true prevalence rate of MLD is unknown but is estimated to be between 1 in 40,000 and 1 in 160,000.
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As per the MalaCards Human Disease Database, MLD is inherited in an autosomal recessive manner with a prevalence of 1-9 per 100,000 in Europe, the United States, and worldwide and the age of onset of different forms of MLD varies.
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According to Natalia et al. study titled “Arylsulfatase A Deficiency” stated that the onset of Late-infantile, Juvenile MLD, and Adult MLD is before age 30 months, between age 30 months and 16 years and after age 16 years respectively.
Metachromatic Leukodystrophy Market
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 Metachromatic Leukodystrophy pipeline therapies. It also thoroughly assesses the Metachromatic Leukodystrophy market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Metachromatic Leukodystrophy 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.
Metachromatic Leukodystrophy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Metachromatic Leukodystrophy epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Metachromatic Leukodystrophy 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 Metachromatic Leukodystrophy Epidemiology, Segmented as –
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Prevalent Population of Metachromatic Leukodystrophy (MLD) in the 7 MM [2019-2032]
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Age-specific Prevalent Population of Metachromatic Leukodystrophy (MLD) in the 7 MM {Late infantile form, Juvenile form, and Adult form} [2019-2032]
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Diagnosed and Treatable Cases of Metachromatic Leukodystrophy (MLD) in the 7 MM [2019-2032]
Metachromatic Leukodystrophy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Metachromatic Leukodystrophy market or expected to be launched during the study period. The analysis covers the Metachromatic Leukodystrophy 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 Metachromatic Leukodystrophy drugs based on their sale and market share.
The report also covers the Metachromatic Leukodystrophy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Metachromatic Leukodystrophy 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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Metachromatic Leukodystrophy Therapeutics Analysis
Currently, the market holds no effective treatment to reverse the deterioration and loss of function that MLD causes. Treatment for the condition focuses on managing symptoms and improving your quality of life i.e. it’s generally supportive in nature. The management of MLD can be done by using medications, physical, occupational, and speech therapy, and nutritional assistance can help the patients suffering from MLD. Bone marrow transplantation may delay the progression of the disease in some infantile-onset cases.
Medications, mainly painkillers, can be used which may reduce the signs and symptoms and thereby relieve muscle pain. The signs and symptoms of MLD that can be resolved using medicines include such as behavioral problems, seizures, difficulty with sleeping, gastrointestinal issues, infection, and pain. Physical therapy can help by curing stiffness in the joints to get short-term relief. One can rely on a wheelchair, walker, or other assistive devices as the condition progresses.
To further improve the treatment outlook, several major pharma and biotech companies are developing therapies for Metachromatic Leukodystrophy. Currently, Orchard Therapeutics is leading the therapeutics market with its Metachromatic Leukodystrophy drug candidates in the most advanced stage of clinical development.
Metachromatic Leukodystrophy Companies Actively Working in the Therapeutics Market Include
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Takeda
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Denali Therapeutics
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Orchard Therapeutics
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Homology Medicines
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Passage Bio
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ArmaGen Technologies
And Many Others
Emerging and Marketed Metachromatic Leukodystrophy Therapies Covered in the Report Include:
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OTL-200: Orchard Therapeutics
OTL-200 (autologous CD34+ cell-enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), referred to as Libmeldy in the European Union, is an ex vivo autologous hematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020. OTL-200 is an investigational therapy that has not been approved by the U.S. Food and Drug Administration (FDA) or any other health authority.
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TAK-611: Takeda
TAK-611, a recombinant cerebroside sulfatase is being developed by Takeda as an enzyme replacement therapy for the treatment of metachromatic leukodystrophy (MLD), a rare hereditary disease characterized by the accumulation of toxic lipids that destroy the myelin sheath surrounding neurons. It is currently in the phase II stage of development for metachromatic leukodystrophy.
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Metachromatic Leukodystrophy Competitive Intelligence Analysis
4. Metachromatic Leukodystrophy Market Overview at a Glance
5. Metachromatic Leukodystrophy Disease Background and Overview
6. Metachromatic Leukodystrophy Patient Journey
7. Metachromatic Leukodystrophy Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Metachromatic Leukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Metachromatic Leukodystrophy Unmet Needs
10. Key Endpoints of Metachromatic Leukodystrophy Treatment
11. Metachromatic Leukodystrophy Marketed Therapies
12. Metachromatic Leukodystrophy Emerging Drugs and Latest Therapeutic Advances
13. Metachromatic Leukodystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Metachromatic Leukodystrophy Market Outlook (In US, EU5, and Japan)
16. Metachromatic Leukodystrophy Companies Active in the Market
17. Metachromatic Leukodystrophy Access and Reimbursement Overview
18. KOL Views on the Metachromatic Leukodystrophy Market
19. Metachromatic Leukodystrophy Market Drivers
20. Metachromatic Leukodystrophy 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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