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Adrenoleukodystrophy Market Insights, Epidemiology and Market Forecast till 2030 by DelveInsight

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Adrenoleukodystrophy Market Insights, Epidemiology and Market Forecast till 2030 by DelveInsight

September 11
17:35 2020
Adrenoleukodystrophy Market Insights, Epidemiology and Market Forecast till 2030 by DelveInsight

DelveInsight Business Research LLP
DelveInsight’s “Adrenoleukodystrophy – Market Insights, Epidemiology, and Market Forecast-2030” report delivers an in-depth understanding of the Adrenoleukodystrophy , historical and forecasted epidemiology as well as the Adrenoleukodystrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very long chain fatty acids (VLCFAs) in the brain. When VLCFAs accumulate, they destroy the protective myelin sheath around nerve cells, responsible for brain function. Without the myelin sheath, the nerves can no longer relay information to and from the brain.

The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addison’s disease and — the most common and most devastating form — cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years. There are three distinct types of X-linked adrenoleukodystrophy: a childhood cerebral form, an adrenomyeloneuropathy type, and a form called Addison disease only.

DelveInsight’s “Adrenoleukodystrophy – Market Insights, Epidemiology, and Market Forecast-2030 report delivers an in-depth understanding of the Adrenoleukodystrophy , historical and forecasted epidemiology as well as the Adrenoleukodystrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Adrenoleukodystrophy market report provides current treatment practices, emerging drugs, Adrenoleukodystrophy market share of the individual therapies, current and forecasted Adrenoleukodystrophy market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Adrenoleukodystrophy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.

Geography Covered

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

Study Period: 2017-2030

View report: https://www.delveinsight.com/sample-request/adrenoleukodystrophy-market

Adrenoleukodystrophy Disease Understanding and Treatment Algorithm

Adrenoleukodystrophy Overview

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. The accumulation of VLCFAs leads to the deterioration of the myelin sheath, and without the sheath, neurons cannot conduct action potentials, this leads to the development of seizures and hyperactivity. Currently, no drug is approved for the treatment of Adrenoleukodystrophy and the available treatment options include hormone replacement therapy, dietary therapy with Lorenzo’s oil and Hematopoietic stem cell transplantation, using either umbilical cord or bone marrow stem cells.

Signs and Symptoms

Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Most people with the adrenomyeloneuropathy type also have adrenocortical insufficiency. In some severely affected individuals, damage to the brain and nervous system can lead to early death.

Causes

ALD is an X-linked recessive condition caused by a mutation in the ABCD1 gene on the X chromosome. Because a female has two X chromosomes, if she inherits the faulty gene, then she still has another X chromosome to offset the mutation. However, because males only have one X chromosome, the gene abnormality causes the disease. With each pregnancy, female ALD carriers have a 25 percent (1 in 4) chance of having a carrier daughter and a 25 (1 in 4) percent chance of having a son affected with the disease.

Adrenoleukodystrophy Epidemiology  

The Adrenoleukodystrophy epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Population of Adrenoleukodystrophy in the 7MM, Gender-specific Prevalence of Adrenoleukodystrophy in the 7MM, Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in the 7MM, and Type-specific Diagnosed Prevalence of ALD in Males in the 7MM. The epidemiology section covers the 7MM countries, the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from the year 2017 to 2030.

Adrenoleukodystrophy Drug Chapters

Lenti D: Bluebird Bio

Lenti D is an investigational gene therapy that is in Phase II/III stage of development for the treatment of cerebral ALD (CALD). Currently, bluebird bio is enrolling patients for a Phase III study (ALD-104) designed to assess the efficacy and safety of Lenti-D after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Additionally, the company is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have participated in ALD-102 and were treated with Lenti-D.

Leriglitazone: Minoryx Therapeutics, S.L.

Leriglitazone (MIN-102) is a metabolite of pioglitazone which shows an excellent brain penetration and safety profile, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones. The drug candidate is in Phase II/III stage of development for the treatment of both cerebral ALD and Adrenomyeloneuropathy (AMN).

Products detail in the report…

Adrenoleukodystrophy Market Outlook

The Adrenoleukodystrophy market outlook of the report helps to build a detailed comprehension of the historic, current and forecasted Adrenoleukodystrophy market trends by analyzing the impact of current and emerging therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a thorough detail of Adrenoleukodystrophy market trend of each current available and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, Adrenoleukodystrophy market in 7MM is expected to change in the study period 2017–2030.

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

Table of contents:

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

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

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

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

Download report: https://www.delveinsight.com/sample-request/adrenoleukodystrophy-market

About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.

Contact us:
Shruti Thakur
[email protected]
+919650213330

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: 9193216187
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City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/

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