Adrenoleukodystrophy (ALD) is an X-linked recessive genetic disorder caused by the abnormality in the ABCD1 gene present on the X chromosome that leads to the accumulation of very-long-chain fatty acids (VLCFAs) in the brain, nervous system, and adrenal gland.
Epidemiology Insights
- Total prevalent population of ALD in the 7MM in 2017 was 55,242
- The prevalent population of ALD in the US was 19,371 in 2017
- Among the EU5, Germany had the highest diagnosed prevalent population of ALD with 1,699 cases
- The highest diagnosed prevalence of ALD was in the United States in 2017 was 4,885
Adrenoleukodystrophy Market Size in the United States in 2017 was USD 523.26 million
Scope of the Report
- The report covers the descriptive overview of Adrenoleukodystrophy , explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
- Comprehensive insight has been provided into the Adrenoleukodystrophy epidemiology and treatment in the 7MM
- Additionally, an all-inclusive account of both the current and emerging therapies for Adrenoleukodystrophy are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
- A detailed review of Adrenoleukodystrophy market; historical and forecasted is included in the report, covering drug outreach in the 7MM
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Adrenoleukodystrophy market
Adrenoleukodystrophy Market Drivers
- Increased number of potential therapies in the pipeline
- Awareness about newborn screening programs
- Increase in the number of Childhood Cerebral Adrenoleukodystrophy (CALD) cases
Adrenoleukodystrophy Emerging Therapies
- Lenti D: bluebird bio
- Leriglitazone: Minoryx Therapeutics, S.L.
- MD1003: MedDay Pharmaceuticals
- MGTA-456: Magenta Therapeutics
- OP-101: Orpheris Inc.
- SOM1201: SOM Biotech
1. Key Insights
2. Adrenoleukodystrophy (ALD): Market Overview at a Glance
2.1. Total Market Share (%) Distribution of ALD in 2017
2.2. Total Market Share (%) Distribution of ALD in 2030
3. Adrenoleukodystrophy: Disease Background and Overview
3.1. Introduction
3.2. Clinical aspects of ALD
3.3. Causes of ALD
3.4. Inheritance pattern of ALD
3.5. ALD-Symptoms
3.6. Pathogenesis and Pathophysiology of ALD
3.7. Diagnosis
3.7.1. ALD Newborn Screening
3.7.2. Tools for evaluating Cerebral ALD
3.7.3. Diagnostic algorithm
3.7.4. HSCT and presymptomatic diagnosis of ALD in Japan
4. Epidemiology and Patient Population
4.1. Key Findings
4.2. Epidemiology Methodology
4.3. KOL Views
4.4. Total Prevalent Population of Adrenoleukodystrophy in the 7MM
5. United States Epidemiology
5.1. Assumptions and Rationale
5.2. Total Prevalent Population of Adrenoleukodystrophy (ALD) in the United States
5.3. Gender-specific Prevalence of Adrenoleukodystrophy in the United States
5.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in the United States
5.5. Type-specific Diagnosed Prevalence of ALD in Males in the United States
6. EU5 Epidemiology
6.1. Germany Epidemiology
6.1.1. Assumptions and Rationale
6.1.2. Total Prevalent Population of Adrenoleukodystrophy in Germany
6.1.3. Gender-specific Prevalence of Adrenoleukodystrophy in Germany
6.1.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in Germany
6.1.5. Type-specific Diagnosed Prevalence of ALD in males in Germany
6.2. France Epidemiology
6.2.1. Assumptions and rationale
6.2.2. Total Prevalent Population of Adrenoleukodystrophy in France
6.2.3. Gender-specific Prevalence of Adrenoleukodystrophy in France
6.2.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in France
6.2.5. Type-specific Diagnosed Prevalence of ALD in Males in France
6.3. Italy Epidemiology
6.3.1. Assumptions and Rationale
6.3.2. Total Prevalent Population of Adrenoleukodystrophy in Italy
6.3.3. Gender-specific Prevalence of Adrenoleukodystrophy in Italy
6.3.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in Italy
6.3.5. Type-specific Diagnosed Prevalence of ALD in Males in Italy
6.4. Spain Epidemiology
6.4.1. Assumptions and Rationale
6.4.2. Total Prevalent Population of Adrenoleukodystrophy in Spain
6.4.3. Gender-specific Prevalence of Adrenoleukodystrophy in Spain
6.4.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in Spain
6.4.5. Type-specific Diagnosed Prevalence of ALD in Males in Spain
6.5. United Kingdom Epidemiology
6.5.1. Assumptions and Rationale
6.5.2. Total Prevalent Population of Adrenoleukodystrophy in the United Kingdom
6.5.3. Gender-specific Prevalence of Adrenoleukodystrophy in the United Kingdom
6.5.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in the United Kingdom
6.5.5. Type-specific Diagnosed Prevalence of ALD in Males in the United Kingdom
7. Japan Epidemiology
7.1. Assumptions and Rationale
7.2. Total Prevalent Population of Adrenoleukodystrophy in Japan
7.3. Gender-specific Prevalence of Adrenoleukodystrophy in Japan
7.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in Japan
7.5. Type-specific Diagnosed Prevalence of ALD in Males in Japan
8. Current Treatment and Medical Practices
8.1. Subtype-specific Treatment
8.2. Treatment Guidelines
8.2.1. Recommendations for Treatment of X-linked ALD in the United States
8.2.2. Adrenoleukodystrophy treatment: European Leukodystrophies Association
8.3. Early intervention and long-term follow-up system for presymptomatic ALD patients in Japan
9. Unmet Needs
10. Emerging Therapies
10.1. Lenti D: bluebird bio
10.1.1. Drug Description
10.1.2. Regulatory Milestone
10.1.3. Other Development Activities
10.1.4. Clinical Development
10.1.5. Safety and Efficacy
10.1.6. Advantages and Disadvantages
10.1.7. Product Profile
10.2. Leriglitazone: Minoryx Therapeutics, S.L.
10.2.1. Drug Description
10.2.2. Regulatory Milestones
10.2.3. Other Development Activities
10.2.4. Clinical Development
10.2.5. Safety and Efficacy
10.2.6. Advantages and Disadvantages
10.2.7. Product Profile
10.3. MD1003: MedDay Pharmaceuticals
10.3.1. Drug Description
10.3.2. Other Development Activities
10.3.3. Clinical Development
10.3.4. Safety and Efficacy
10.3.5. Advantages and Disadvantages
10.3.6. Product Profile
10.4. MGTA-456: Magenta Therapeutics
10.4.1. Product Description
10.4.2. Other Development Activities
10.4.3. Clinical Development
10.4.4. Clinical Trial Update
10.4.5. Safety and Efficacy
10.4.6. Product Profile
10.5. OP-101: Orpheris Inc.
10.5.1. Drug Description
10.5.2. Regulatory Milestones
10.5.3. Other Development Activities
10.5.4. Clinical Development
10.5.5. Product Profile
10.6. SOM1201: SOM Biotech
10.6.1. Product Description
10.6.2. Other Development Activities
10.6.3. Product Profile
11. Failed Therapy
11.1. NV1205: NeuroVia/Archer Daniels Midland
11.1.1. Drug Description
11.1.2. Other Development Activities
11.1.3. Clinical Development
11.1.4. Safety and Efficacy
11.1.5. Advantages and Disadvantages
11.1.6. Product Profile
12. Adrenoleukodystrophy (ALD): 7MM Market Analysis
12.1. Key Findings
12.2. Market Methodology
12.3. Market Size of Adrenoleukodystrophy in the 7MM
12.4. Market Size of Adrenoleukodystrophy by Therapies in the 7MM
13. 7MM: Market Outlook
14. United States
14.1. United States Market Size
14.1.1. Total Market size of Adrenoleukodystrophy in the United States
14.1.2. Market Size of Adrenoleukodystrophy by Therapies in the US
15. EU-5 countries
15.1. Germany Market Size
15.1.1. Total Market size of Adrenoleukodystrophy in Germany
15.1.2. Market Size of Adrenoleukodystrophy by therapies in Germany
15.2. France Market Size
15.2.1. Total Market size of Adrenoleukodystrophy in France
15.2.2. Market Size of Adrenoleukodystrophy by therapies in France
15.3. Italy Market Size
15.3.1. Total Market size of Adrenoleukodystrophy in Italy
15.3.2. Market Size of Adrenoleukodystrophy by therapies in Italy
15.4. Spain Market Size
15.4.1. Total Market size of Adrenoleukodystrophy in Spain
15.4.2. Market Size of Adrenoleukodystrophy by therapies in Spain
15.5. United Kingdom Market Size
15.5.1. Total Market size of Adrenoleukodystrophy in the United Kingdom
15.5.2. Market Size of Adrenoleukodystrophy by therapies in the UK
16. Japan
16.1. Japan Market Size
16.1.1. Total Market size of Adrenoleukodystrophy in Japan
16.1.2. Market Size of Adrenoleukodystrophy by therapies in Japan
17. Market Drivers
18. Market Barriers
19. Appendix
19.1. Bibliography
19.2. Report Methodology
20. DelveInsight Capabilities
21. Disclaimer
22. About DelveInsight
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