Metachromatic Leukodystrophy Market Insights and Treatment Market by DelveInsight

April 23 19:19 2021
Metachromatic Leukodystrophy Market Insights and Treatment Market by DelveInsight

 

Metachromatic Leukodystrophy Market” report has been added to DelveInsight

 

Metachromatic Leukodystrophy disease 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).

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Metachromatic Leukodystrophy Market: Geography Covered

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

 

Metachromatic Leukodystrophy Market Companies

  • Orchard Therapeutics
  • Takeda
  • Magenta Therapeutics

 

Metachromatic Leukodystrophy Drugs

  • OTL-200
  • TAK-611
  • MGTA-456

 

Metachromatic Leukodystrophy Market Report

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.

 

Metachromatic Leukodystrophy Market Research

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 Market Report Highlights

  • According to the United Leukodystrophy Foundation, MLD is inherited in an autosomal recessive manner, and is most commonly caused by a mutation in a gene called Arylsulfatase A (ASA), also called sulfatide sulfatase. The protein produced by ASA is present in the lysosomal compartment of the cell.
  • 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.

 

Metachromatic Leukodystrophy Market Insights

  • As per the Myriad Women’s Health, MLD is divided into three forms infantile (early-onset), juvenile (late-onset), and adult (late-onset) out of which infantile form is the most common with 50 to 60% of all the cases.
  • According to Natalia et al. study titled as “Arylsulfatase A Deficiency” stated that the onset of the 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. Additionally, late-infantile MLD comprises 50 to 60% of affected individuals, juvenile MLD comprises approximately 20 to 30% and adult MLD affects approximately 15 to 20% of MLD patients.

 

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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. Unmet Needs

10. Key Endpoints of Metachromatic Leukodystrophy (MLD) Treatment

11. Marketed Products

12. 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

 

In conclusion, this report provides information such as causes, symptoms, treatment, and Market report, Market Research, Market Drivers, Market Barriers and SWOT Analysis are also incorporated in the report

 

 

 

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