DelveInsight added a report on the “Metachromatic leukodystrophy Market“. It covers an overview, its causes, signs and symptoms, and therapies. Major players are involved in developing therapies for Metachromatic leukodystrophy. The launch of emerging therapies will significantly impact the Metachromatic leukodystrophy market.
Metachromatic leukodystrophy Overview
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).
Some of the key highlights of the Metachromatic Leukodystrophy Market Report
- The prognosis for MLD is poor. Most children within the infantile form die by age 5. Symptoms of the juvenile form progress with death occurring 10 to 20 years following onset. Those persons affected by the adult form typically die within 6 to 14 years following onset of symptoms.
- The true prevalence rate of MLD is unknown, but is estimated to be between 1 in 40,000 and 1 in 160,000.
- Through various secondary studies it can be concluded that MLD affects both males and females equally.
- Metachromatic Leukodystrophy companies included such as Orchard Therapeutics, Takeda, Magenta Therapeutics, and several others.
- Metachromatic Leukodystrophy drugs included such as OTL-200, TAK-611, MGTA-456, and several others.
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Metachromatic Leukodystrophy Symptoms
There are three types of MLD based on the age symptoms appear: late-infantile MLD, juvenile MLD, and adult MLD. All subtypes ultimately affect both intellectual and motor function. Symptoms vary by type but can include ataxia, behavioural abnormality, behavioural changes, coma, decreased nerve conduction velocity, intellectual disability, muscle weakness, neurological speech impairment, seizures, joint stiffness, reduced tendon reflexes, spasticity, abnormal muscle movements, difficulty walking, irritability, swallowing difficulty, etc.
Metachromatic Leukodystrophy Cause
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 centres. 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.
Get in-depth information about Metachromatic Leukodystrophy Market Size
Metachromatic Leukodystrophy Market Insights
Expected launch of potential therapies may increase market size in the coming years, assisted by an increase in prevalent population of MLD. Owing to the positive outcomes of the several drug candidates during the developmental stage by key players such as Orchard Therapeutics (OTL-200), Takeda (TAK-611), Magenta Therapeutics (MGTA-456) and others have the potential to create a significant positive shift in the MLD Market Size. However, the current treatment paradigm of MLD includes use of medications, physical therapy, occupational and speech therapy, nutritional assistance and bone marrow transplantation.
Metachromatic Leukodystrophy Treatment
- Medications (Pain Killers)
- Nutritional Assistance
- Bone Marrow Transplantation
- Physical Therapy
- Potential Therapies (Enzyme Replacement Therapy and Gene Therapy)
- Occupational & Speech Therapy
Metachromatic Leukodystrophy Market Insights
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 progression of the disease in some infantile-onset cases.
Metachromatic Leukodystrophy Emerging Therapy Assessment
The pipeline of Metachromatic Leukodystrophy (MLD) possess few potential key players, such as Takeda, Orchard Therapeutics and Magenta Therapeutics. The dynamics of MLD market is anticipated to change in the coming years owing to the improvement in the research and development activities so that market will comprise of efficient treatment options. The launch of emerging therapies is expected during the forecast period of 2020-2030.
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Table of content
1. Key Insights
2. Executive Summary of Metachromatic Leukodystrophy (MLD)
3. Competitive Intelligence Analysis for Metachromatic Leukodystrophy (MLD)
4. Metachromatic Leukodystrophy (MLD): Market Overview at a Glance
5. Metachromatic Leukodystrophy (MLD): Disease Background and Overview
6. Patient Journey
7. Metachromatic Leukodystrophy (MLD) Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Metachromatic Leukodystrophy (MLD) Unmet Needs
10. Key Endpoints of Metachromatic Leukodystrophy (MLD) Treatment
11. Metachromatic Leukodystrophy (MLD) Marketed Products
12. Metachromatic Leukodystrophy (MLD) Emerging Therapies
13. Metachromatic Leukodystrophy (MLD): Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Metachromatic Leukodystrophy (MLD)
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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