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异染性脑白质营养不良治疗的全球市场（2022-2029）
市场调查报告书
商品编码
1082815

异染性脑白质营养不良治疗的全球市场（2022-2029）

Global Metachromatic leukodystrophy Treatment Market - 2022-2029
出版日期: | 出版商: DataM Intelligence | 英文 160 Pages | 商品交期: 约2个工作天内

价格

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简介目录

在本报告中，我们调查和分析了世界上异染性白色营养不良的治疗，以及市场概况、市场趋势、市场促进/抑制因素、COVID-19 的影响、行业分析、细分市场/地区，提供分析、竞争形势、企业概况等资讯。

目录

第 1 章全球变态脑白质营养不良治疗市场：范围和研究方法

  • 调查方法
  • 调查的目的和范围

第 2 章全球变态脑白质营养不良治疗市场：市场定义和概述

第 3 章全球变态脑白质营养不良治疗市场：执行摘要

  • 按类型划分的市场细分
  • 按诊断划分的市场细分
  • 按治疗类型划分的市场细分
  • 按最终用户划分的市场细分
  • 按地区划分的市场细分

第 4 章全球变态脑白质营养不良治疗市场机制

  • 影响市场的因素
    • 促进因素
    • 抑制因素
    • 市场机会
    • 影响分析

第 5 章全球变态脑白质营养不良治疗市场：行业分析

  • 供应链分析
  • 价格分析
  • 未满足的需求

第 6 章全球变态脑白质营养不良治疗市场：COVID-19 分析

  • 分析 COVID-19 对市场的影响
    • 之前的 COVID-19 市场情景
    • 当前 COVID-19 市场情况
    • COVID-19 后或未来情景
  • COVID-19 的价格波动
  • 供需范围
  • 大流行期间与市场相关的政府工作
  • 製造商的战略努力
  • 结论

第 7 章全球脑白质营养不良治疗市场：按类型

  • 简介
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场吸引力指数：按类型
  • 晚期婴儿脑白质营养不良
    • 简介
    • 市场规模分析/同比增长分析（%）：2020-2029和2021-2029（100万美元）
  • 幼年同种异体脑白质营养不良
  • 成人脑白质营养不良

第 8 章全球脑白质营养不良治疗市场：通过诊断

  • 简介
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场吸引力指数：按类型
  • 验血
    • 简介
    • 市场规模分析/同比增长分析（%）：2020-2029和2021-2029（100万美元）
  • 基因检测
  • 神经传导研究
  • 磁共振成像 (MRI)
  • 心理和认知测试
  • 其他

第 9 章全球脑白质营养不良治疗市场：按治疗类型

  • 简介
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场吸引力指数：按治疗类型
  • 药物治疗
    • 简介
    • 市场规模分析/同比增长分析（%）：2020-2029和2021-2029（100万美元）
  • 基因治疗
    • AGT-183
    • GSK-2696274
    • 其他
  • □替代疗法或强化疗法
  • 底物还原疗法
  • 其他

第 10 章全球脑白质营养不良治疗市场：按最终用户

  • 简介
    • 市场规模分析/同比增长分析（%）：按最终用户
    • 市场吸引力指数：按最终用户分类
  • 医院
    • 简介
    • 市场规模分析/同比增长分析（%）：2020-2029和2021-2029（100万美元）
  • 诊所
  • 诊断中心
  • 其他

第 11 章全球脑白质营养不良治疗市场：按地区

  • 简介
    • 区域市场规模分析/同比增长分析（%）：2020-2029和2021-2029（100万美元）
    • 各地区市场吸引力指数
  • 北美
    • 简介
    • 特定地区的主要动态
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场规模分析/同比增长分析（%）：通过诊断
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场规模分析/同比增长分析（%）：按最终用户
    • 市场规模分析/同比增长分析 (%)：按国家/地区
  • 欧洲
    • 简介
    • 特定地区的主要动态
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场规模分析/同比增长分析（%）：通过诊断
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场规模分析/同比增长分析（%）：按最终用户
    • 市场规模分析/同比增长分析 (%)：按国家/地区
  • 南美洲
    • 简介
    • 特定地区的主要动态
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场规模分析/同比增长分析（%）：通过诊断
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场规模分析/同比增长分析（%）：按最终用户
    • 市场规模分析/同比增长分析 (%)：按国家/地区
  • 亚太地区
    • 简介
    • 特定地区的主要动态
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场规模分析/同比增长分析（%）：通过诊断
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场规模分析/同比增长分析（%）：按最终用户
    • 市场规模分析/同比增长分析 (%)：按国家/地区
  • 中东和非洲
    • 简介
    • 特定地区的主要动态
    • 市场规模分析/同比增长分析（%）：按类型
    • 市场规模分析/同比增长分析（%）：通过诊断
    • 市场规模分析/同比增长分析（%）：按治疗类型
    • 市场规模分析/同比增长分析（%）：按最终用户

第 12 章全球变态脑白质营养不良治疗市场：竞争形势

  • 主要发展及战略
  • 企业份额分析
  • 产品基准
  • 知名大公司名单

第 13 章全球变态脑白质营养不良治疗市场：公司简介

  • ArmaGen, Inc.
    • 公司简介
    • 产品组合/描述
    • 主要亮点
    • 财务概览
  • GlaxoSmithKline plc
  • Takeda Pharmaceutical Company Limited
  • Orchard Therapeutics plc
  • Magenta Therapeutics
  • Recursion Pharmaceuticals Inc.
  • RegenxBio Inc.

第 14 章全球脑白质营养不良治疗市场：DataM

简介目录
Product Code: DMPH5227

Market Overview

The global metachromatic leukodystrophy treatment market size was valued US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Metachromatic leukodystrophy is a rare hereditary (genetic) condition characterized by the accumulation of fatty substances (lipids) in cells, mainly in the brain, spinal cord, and peripheral nerves. A lack of an enzyme that helps break down lipids called sulfatides causes this accumulation. Because the substance that covers and protects nerve cells (myelin) is destroyed, the brain and nervous system gradually lose function. Metachromatic leukodystrophy is currently incurable. Early detection and treatment may help manage some signs and symptoms and postpone the progression of the illness, depending on the form and age of start.

Market Dynamics

Innovation in the products for treating demyelinating conditions is expected to drive market growth.

Joanne Kurtzberg an American professor of pediatrics and pathology at Duke University has developed a product called Duke O Cell or DUOC-01, which is a cell generated from umbilical cord blood that has been demonstrated to help people with demyelinating diseases. It's been used as a bridge therapy to help youngsters with inherited metabolic problems engraft faster. Moreover, Atidarsagene autotemcel (arsa-cel) is a gene therapy medicinal product based on autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding the ARSA cDNA driving supranormal ARSA expression in HSPCs and their progeny, with preliminary evidence of safety and efficacy in patients with early-onset MLD. Thus, from the above statements, the market is expected to drive in the forecast period.

Restraint:

The high cost of therapies for the treatment of Metachromatic leukodystrophy and there is no single FDA-approved pharmacological product on the market that can completely cure metachromatic leukodystrophy are the factors the market is expected to hamper in the forecast period.

COVID-19 Impact Analysis

The COVID-19 pandemic has moderately impacted healthcare systems and the market. Standard operating procedures for Personal Protective Equipment (PPE) for home care nurses were unclear during the pandemic. The doctors contacted all patients by email or telephone, as well as sent letters to those who did not have access to email or the internet. With little evidence of the impact of SARS-CoV-2 infections on rare diseases early in the pandemic, we decided that the danger of developing SARS-CoV-2 outweighed the risk of treatment interruption. Moreover, disruptions in therapy, whether planned or inevitable, resulted in a deterioration of clinical parameters and quality of life, as well as potentially negative outcomes such as cardiac events and/or renal function decline.

Additionally, the severity of the impact of the COVID-19 pandemic on business, including on preclinical and clinical development, its supply chain, and commercial programs got affected. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.

Segment Analysis

Gene Therapy segment is expected to hold the largest market share in metachromatic leukodystrophy treatment market

The gene therapy segment is expected to dominate in 2021. The segment's growth is beneficial because gene therapy showed benefits in pediatric patients with early-onset metachromatic leukodystrophy (MLD). There is currently no cure for MLD. In contrast, the European Medicines Agency (EMA) has recommended that the gene therapy Libmeldy, which consists of cryopreserved autologous CD34+ cells encoding the arylsulfatase-A gene, be given marketing authorization in the European Union to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is for children who have been diagnosed as carriers of the faulty gene but have not yet shown symptoms and have the 'late infantile' or 'early juvenile' variants of MLD. It is used in children who have been diagnosed with the early juvenile form of the disease and have begun to show symptoms while still being able to walk independently and before cognitive impairment. Thus, from the above statements, the market segment is expected to hold the largest market share in the forecast period.

Geographical Analysis

North America region holds the largest market share in the global metachromatic leukodystrophy treatment market

In 2021, North America accounted for the highest revenue share. The increasing prevalence of metachromatic leukodystrophy, increased number of diagnosed patients, ongoing clinical trials, rise in government funding for research & development for the treatment, and launch of contact registry by the organizations in the region are some of the factors the market is expected to boost in the forecast period. For instance, metachromatic leukodystrophy ranges from 1/40,000 births in the United States of America. Moreover, The National Organization for Rare Disorders, Inc. has initiated a large-scale study to investigate Metachromatic Leukodystrophy (MLD), a brain disease that causes irreparable white matter destruction. The Metachromatic Leukodystrophy Community United Research and Engagement Survey (MLDCures) is a new project that provides a platform for Metachromatic Leukodystrophy patients worldwide to contribute information about the disease (MLD). Its goal is to create an international resource that scientists can use in future research. Additionally, Cure MLD is collaborating on the study with the National Organization for Rare Disorders (NORD), an independent organization that developed the IAMRARE platform to aid in the identification and treatment of all 7,000 rare diseases. Thus, from the above statements, the North American region is expected to hold the largest market share in the forecast period.

Competitive Landscape

Major key players in the metachromatic leukodystrophy treatment market are ArmaGen, Inc., GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, Orchard Therapeutics plc, Magenta Therapeutics, Recursion Pharmaceuticals Inc. and RegenxBio Inc.

ArmaGen, Inc.:

Overview:

ArmaGen is a privately held, clinical-stage biotechnology company, with headquarters and research operations located in Calabasas, CA focused on developing revolutionary therapies for severe neurological disorders. The company is advancing innovative therapies for the treatment of currently unaddressed neurological complications of lysosomal storage disorders (LSDs), as well as neurodegenerative diseases such as Alzheimer's and Parkinson's. Moreover, ArmaGen has eight compounds in development. The company's lead product candidates are AGT-182 for Hunter syndrome, which is in Phase 1 clinical trials and AGT-181 for Hurler syndrome, which is in Phase 2 trials. The company is advancing its pipeline through licensing and collaboration agreements, in-house development efforts, and future partnering opportunities.

­Product Portfolio:

AGT-183: AGT-183 is an investigational enzyme replacement therapy (ERT) for the treatment of metachromatic leukodystrophy (MLD), a lysosomal storage disease that arises from a deficiency of the enzyme arylsulfatase (ASA).

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The global metachromatic leukodystrophy treatment market report would provide an access to an approx. 45+market data table, 40+figures and 160pages.

Target Audience

Service Providers/ Buyers

Industry Investors/Investment Bankers

Education & Research Institutes

Research Professionals

Emerging Companies

Manufacturers

Table of Contents

1. Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Market Definition and Overview

3. Executive Summary

  • 3.1. Market Snippet by Type
  • 3.2. Market Snippet by Diagnosis
  • 3.3. Market Snippet by Treatment Type
  • 3.4. Market Snippet by End user
  • 3.5. Market Snippet by Region

4. Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Innovation in the products for treating demyelinating conditions is expected to drive market growth.
    • 4.1.2. Restraints:
      • 4.1.2.1. The high cost of therapies for the treatment of Metachromatic leukodystrophy is expected to hamper the market growth in the forecast period.
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Supply Chain Analysis
  • 5.2. Pricing Analysis
  • 5.3. Unmet Needs

6. COVID-19 Analysis

  • 6.1. Analysis of Covid-19 on the Market
    • 6.1.1. Before COVID-19 Market Scenario
    • 6.1.2. Present COVID-19 Market Scenario
    • 6.1.3. After COVID-19 or Future Scenario
  • 6.2. Pricing Dynamics Amid Covid-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturers Strategic Initiatives
  • 6.6. Conclusion

7. By Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 7.1.2. Market Attractiveness Index, By Type Segment
  • 7.2. Late-Infantile MLD*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 7.3. Juvenile MLD
  • 7.4. Adult MLD

8. By Diagnosis

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 8.1.2. Market Attractiveness Index, By Diagnosis Segment
  • 8.2. Blood tests*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 8.3. Genetic tests
  • 8.4. Nerve conduction study
  • 8.5. Magnetic resonance imaging (MRI)
  • 8.6. Psychological and cognitive tests
  • 8.7. Others

9. By Treatment Type

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.1.2. Market Attractiveness Index, By Treatment Type Segment
  • 9.2. Pharmacotherapy*
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 9.3. Gene Therapy
    • 9.3.1. AGT-183
    • 9.3.2. GSK-2696274
    • 9.3.3. Others
  • 9.4. Enzyme replacement or enhancement therapy
  • 9.5. Substrate reduction therapy
  • 9.6. Others

10. By End user

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
    • 10.1.2. Market Attractiveness Index, By End user Segment
  • 10.2. Hospital*
    • 10.2.1. Introduction
    • 10.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 10.3. Clinics
  • 10.4. Diagnostic Centers
  • 10.5. Others

11. By Region

  • 11.1. Introduction
    • 11.1.1. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
    • 11.1.2. Market Attractiveness Index, By Region
  • 11.2. North America
    • 11.2.1. Introduction
    • 11.2.2. Key Region-Specific Dynamics
    • 11.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 11.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 11.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
    • 11.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.2.7.1. U.S.
      • 11.2.7.2. Canada
      • 11.2.7.3. Mexico
  • 11.3. Europe
    • 11.3.1. Introduction
    • 11.3.2. Key Region-Specific Dynamics
    • 11.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 11.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 11.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
    • 11.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.3.7.1. Germany
      • 11.3.7.2. U.K.
      • 11.3.7.3. France
      • 11.3.7.4. Italy
      • 11.3.7.5. Spain
      • 11.3.7.6. Rest of Europe
  • 11.4. South America
    • 11.4.1. Introduction
    • 11.4.2. Key Region-Specific Dynamics
    • 11.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 11.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 11.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
    • 11.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.4.7.1. Brazil
      • 11.4.7.2. Argentina
      • 11.4.7.3. Rest of South America
  • 11.5. Asia Pacific
    • 11.5.1. Introduction
    • 11.5.2. Key Region-Specific Dynamics
    • 11.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 11.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 11.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
    • 11.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.5.7.1. China
      • 11.5.7.2. India
      • 11.5.7.3. Japan
      • 11.5.7.4. Australia
      • 11.5.7.5. Rest of Asia Pacific
  • 11.6. Middle East and Africa
    • 11.6.1. Introduction
    • 11.6.2. Key Region-Specific Dynamics
    • 11.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
    • 11.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 11.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user

12. Competitive Landscape

  • 12.1. Key Developments and Strategies
  • 12.2. Company Share Analysis
  • 12.3. Product Benchmarking
  • 12.4. List of Key Companies to Watch

13. Company Profiles

  • 13.1. ArmaGen, Inc.*
    • 13.1.1. Company Overview
    • 13.1.2. Product Portfolio and Description
    • 13.1.3. Key Highlights
    • 13.1.4. Financial Overview
  • 13.2. GlaxoSmithKline plc
  • 13.3. Takeda Pharmaceutical Company Limited
  • 13.4. Orchard Therapeutics plc
  • 13.5. Magenta Therapeutics
  • 13.6. Recursion Pharmaceuticals Inc.
  • 13.7. RegenxBio Inc.

LIST NOT EXHAUSTIVE

14. Global Metachromatic leukodystrophy Treatment Market - DataM

  • 14.1. Appendix
  • 14.2. About Us and Applications
  • 14.3. Contact Us