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全球脊髓性肌肉萎缩症治疗市场：市场规模、份额和趋势分析（按类型、治疗方法、给药途径、地区和细分，2025-2030 年）

Spinal Muscular Atrophy Treatment Market Size, Share & Trends Analysis Report By Type (Type 1, Type 2, Type 3, Type 4), By Treatment (Gene Therapy, Drug), By Route of Administration, By Region, And Segment Forecasts, 2025 - 2030
出版日期: | 出版商: Grand View Research | 英文 100 Pages | 商品交期: 2-10个工作天内

价格

脊髓性肌肉萎缩症治疗市场的成长与趋势：

根据Grand View Research Inc.的最新研究，全球脊髓性肌肉萎缩症治疗市场规模预计到2030年将达到130.9亿美元，2025年至2030年复合年增长率为18.0%。

这一增长主要得益于产品核准和上市数量的增加。例如，2020年8月，基因泰克开发的Evrysdi（risdiplam）获得食品药物管理局核准，用于治疗2个月及以上的脊髓性肌肉萎缩症（SMA）儿科患者。

此外，2020年5月，诺华公司SMA候选药物Zolgensma（onasemnogene abeparvovec）获得欧盟委员会（EC）有条件核准，用于治疗SMA患者。这是唯一在欧洲核准的基因治疗药物。 2021年3月，PTC Therapeutics获得EMA授予的Evrysdi（risdiplam）上市核准，用于治疗1型、2型和3型脊髓性肌肉萎缩症患者。因此，预计产品核可数量的增加将推动脊髓性肌肉萎缩治疗市场的发展。

诺华公司透过其全球管理存取计画 (MAP) 免费提供药物，为 SMA 患者提供支援。该计划向全球符合条件的 SMA 患者免费提供 100 剂 Zolgenma，包括北美洲和南美洲、亚洲、澳洲、欧洲和非洲。因此，Zolgensma 此类支援计画的存在也有助于加强促销力度并创造收益。

全球新生儿筛检和便利的诊断工具取得应成为常规方法的一部分。这将确保早期诊断并及时治疗。例如，2021 年 7 月，北卡罗来纳州教堂山北卡罗来纳大学医学中心的婴儿夏洛特透过 EarlyCheck新生儿筛检计画被诊断出患有脊髓性肌肉萎缩症 (SMA)，并接受了具有突破性的基因治疗。此类筛检计画有望促进该疾病的早期诊断和治疗。

此外，製药公司正与研究机构进行多项大规模合作，开发治疗脊髓性肌肉萎缩症患者的新治疗方法。例如，2021年3月，PTC Therapeutics, Inc.与脊髓性肌肉萎缩症基金会达成研究合作，以推进科学研究和开发治疗脊髓性肌肉萎缩症患者的新疗法。

然而，治疗相关的高成本以及参与企业数量少可能会在预测期内抑制市场的成长。孤儿药研发高成本是产品价格上涨的主要因素。例如，最常用的药物 Spinraza 每次注射的费用约为 125,000 美元，每年的费用为 750,000 美元。此外，Zolgensma (AVXS-101) 每次治疗的费用为 210 万美元。

脊髓性肌肉萎缩症治疗市场：分析概述

  • 根据类型，1 型脊髓性肌肉萎缩症在 2024 年占据了最大的市场占有率，这归因于 1 型脊髓性肌肉萎缩症患者的高盛行率和治疗产品的广泛可用性。
  • 基于治疗方法，由于市场渗透率的不断提高，预计基因治疗领域在预测期内将增长最快。 Zolgensma 是唯一在 37 个国家核准治疗 SMA 患者的基因疗法。
  • 基于药物，由于优惠的保险报销政策和产品知名度的提高，Spinraza 细分市场将在 2024 年占据市场主导地位。 SPINRAZA 目前在义大利、挪威和荷兰三个欧洲国家已全额报销。
  • 根据给药途径，注射剂在 2024 年占据了市场主导地位，因为透过这种给药途径向 SMA 患者註射药物具有很高的安全性、有效性和耐受性。
  • 预计预测期内亚太地区将成长最快。该地区的成长得益于新产品的进入。例如，2021年7月，罗氏公司在印度推出了Evrysdi（risdiplam），用于治疗脊髓性肌肉萎缩症患者。

目录

第一章 分析方法及范围

第 2 章执行摘要

3. 脊髓性肌肉萎缩症治疗市场：驱动因素、趋势与范围

  • 市场联动展望
  • 市场动态
    • 市场驱动因素分析
    • 市场限制分析
  • 商业环境分析
    • 产业分析：波特五力分析
    • PESTEL 分析

第 4 章脊髓性肌肉萎缩症治疗市场：按类型分類的商业分析

  • 按类型分類的市场占有率（2024 年和 2030 年）
  • 按类型分類的仪表板
  • 按类型分類的市场规模预测及趋势分析（2018-2030）
  • 类型 1
  • 类型 2
  • 类型 3
  • 类型 4

第 5 章脊髓性肌肉萎缩症治疗市场：按治疗方法进行的商业分析

  • 治疗方法的市场占有率（2024 年和 2030 年）
  • 治疗方法仪表板
  • 市场规模预测及治疗方法趋势分析（2018-2030）
  • 基因治疗
  • 药物

6. 脊髓性肌肉萎缩症治疗市场：依给药途径分類的业务分析

  • 按管理途径分類的市场占有率（2024 年和 2030 年）
  • 管理路线仪表板
  • 依管理途径分類的市场规模预测及趋势分析（2018-2030 年）
  • 口服
  • 注射

7. 脊髓性肌肉萎缩症治疗市场：各地区业务分析

  • 按地区分類的市场占有率分析（2024 年和 2030 年）
  • 市场仪表板：按地区
  • 市场规模预测及趋势分析（2018-2030年）
  • 北美洲
    • 按国家/地区，2018 年至 2030 年
    • 美国
    • 加拿大
    • 墨西哥
  • 欧洲
    • 按国家/地区，2018 年至 2030 年
    • 英国
    • 德国
    • 法国
    • 义大利
    • 西班牙
    • 挪威
    • 丹麦
    • 瑞典
  • 亚太地区
    • 日本
    • 中国
    • 印度
    • 韩国
    • 澳洲
    • 泰国
  • 拉丁美洲
    • 按国家/地区，2018 年至 2030 年
    • 巴西
    • 阿根廷
  • 中东和非洲
    • 按国家/地区，2018 年至 2030 年
    • 南非
    • 沙乌地阿拉伯
    • 阿拉伯聯合大公国
    • 科威特

第八章 竞争格局

  • 市场参与企业概况
  • 公司市场地位分析
  • 公司分类
  • 战略地图
  • 公司简介/上市公司
    • Biogen
    • Novartis AG
    • Pfizer Inc.
    • Ionis Pharmaceuticals
    • Biohaven, Ltd.
    • F. Hoffmann-La Roche Ltd
    • Cytokinetics
    • Scholar Rock, Inc.
    • PTC Therapeutics
    • NMD PHARMA A/S
Product Code: GVR-2-68038-626-4

Spinal Muscular Atrophy Treatment Market Growth & Trends:

The global spinal muscular atrophy treatment market size is expected to reach USD 13.09 billion by 2030, registering a CAGR of 18.0% from 2025 to 2030, according to a new study by Grand View Research Inc. This growth can be attributed to the increasing product approval and launches. For instance, in August 2020, the Food and Drug Administration approved Evrysdi (risdiplam) developed by Genentech, Inc., for the treatment of pediatric patients aged two months and older with spinal muscular atrophy (SMA).

In addition, in May 2020, Novartis AG received conditional approval to its SMA candidate Zolgensma (onasemnogene abeparvovec) from the European Commission (EC) to treat patients with SMA. It is only approved gene therapy in Europe. In March 2021, PTC Therapeutics, Inc., received marketing approval for Evrysdi (risdiplam) from the EMA for the treatment of patient with type1, 2, and 3 spinal muscular atrophy. Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.

Novartis AG is supporting SMA patients by offering medicine at free of cost through global Managed Access Program (MAP). This program provides 100 doses of Zolgensma at free of charge to eligible SMA patients across continents including North America, South America, Asia, Australia, Europe, and Africa. Thus, presence of such supportive programs for Zolgensma strengthens promotional activities and also aid in generating revenue.

Global SMA newborn screening and easy access to diagnostic tools should be included in routine approach. This would ensure early diagnosis and timely access to treatment. For instance, in July 2021, a charlotte baby received a breakthrough gene therapy treatment for Spinal Muscular Atrophy (SMA) at UNC Medical Center in Chapel Hill, N.C., after diagnosis with spinal muscular atrophy through Early Check newborn screening program. Such screening programs are expected to boost early diagnosis and treatment of disease.

Moreover, there are several extensive research collaborations undertaken by pharmaceutical companies with research institutes for developing novel therapies for the treatment of spinal muscular atrophy patients. For instance, in March 2021, PTC Therapeutics, Inc., and The Spinal Muscular Atrophy Foundation entered into research collaboration to advance scientific research and developing new treatment to treat patients with SMA.

However, high costs associated with the treatment and low availability of participant for clinical trial purposes may restrain market growth over the forecast period. The high cost related to research and development of an orphan drug is a major factor of rising prices of product. For instance, the most commonly used drug Spinraza costs around USD 125,000 per injection and USD 750,000 annually. Furthermore, Zolgensma (AVXS-101) costs USD 2.1 million for one time treatment.

Spinal Muscular Atrophy Treatment Market Report Highlights:

  • By type, the type-1 segment held the largest market share in 2024 due to higher prevalence and wide availability of products for the treatment of patients with type-1 spinal muscular atrophy
  • By treatment, the gene therapy segment is expected to be the fastest growing segment over the forecast period due to increase market penetration. Zolgensma is the only approved gene therapy in 37 countries to treat patient with SMA
  • By drug, the Spinraza segment dominated the market in 2024 due to presence of supportive reimbursement policies and increasing awareness about products. Currently, Spinraza is applicable for full reimbursement in three European countries Italy, Norway, Netherlands
  • By route of administration, the injection segment dominated the market in 2024 due to high safety, efficacy, and tolerability associated with infusion drugs to SMA patients through this route of administration
  • Asia Pacific is expected to be the fastest growing region during the forecast period. The growth of region is attributable to the entry of new products into the region. For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India

Table of Contents

Chapter 1. Methodology and Scope

  • 1.1. Market Segmentation & Scope
  • 1.2. Segment Definitions
    • 1.2.1. Type
    • 1.2.2. Treatment
    • 1.2.3. Route of Administration
  • 1.3. Estimates and Forecast Timeline
  • 1.4. Research Methodology
  • 1.5. Information Procurement
    • 1.5.1. Purchased Database
    • 1.5.2. GVR's Internal Database
    • 1.5.3. Secondary Sources
    • 1.5.4. Primary Research
  • 1.6. Information Analysis
    • 1.6.1. Data Analysis Models
  • 1.7. Market Formulation & Data Visualization
  • 1.8. Model Details
    • 1.8.1. Commodity Flow Analysis
  • 1.9. List of Secondary Sources
  • 1.10. Objectives

Chapter 2. Executive Summary

  • 2.1. Market Snapshot
  • 2.2. Segment Snapshot
  • 2.3. Competitive Landscape Snapshot

Chapter 3. Spinal Muscular Atrophy Treatment Market Variables, Trends, & Scope

  • 3.1. Market Lineage Outlook
  • 3.2. Market Dynamics
    • 3.2.1. Market Driver Analysis
    • 3.2.2. Market Restraint Analysis
  • 3.3. Business Environment Analysis
    • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
      • 3.3.1.1. Supplier Power
      • 3.3.1.2. Buyer Power
      • 3.3.1.3. Substitution Threat
      • 3.3.1.4. Threat of New Entrants
      • 3.3.1.5. Competitive Rivalry
    • 3.3.2. PESTLE Analysis

Chapter 4. Spinal Muscular Atrophy Treatment Market: Type Business Analysis

  • 4.1. Type Market Share, 2024 & 2030
  • 4.2. Type Segment Dashboard
  • 4.3. Market Size & Forecasts and Trend Analysis, by Type, 2018 to 2030 (USD Billion)
  • 4.4. Type 1
    • 4.4.1. Type 1 Market, 2018 - 2030 (USD Billion)
  • 4.5. Type 2
    • 4.5.1. Type 2 Market, 2018 - 2030 (USD Billion)
  • 4.6. Type 3
    • 4.6.1. Type 3 Market, 2018 - 2030 (USD Billion)
  • 4.7. Type 4
    • 4.7.1. Type 4 Market, 2018 - 2030 (USD Billion)

Chapter 5. Spinal Muscular Atrophy Treatment Market: Treatment Business Analysis

  • 5.1. Treatment Market Share, 2024 & 2030
  • 5.2. Treatment Segment Dashboard
  • 5.3. Market Size & Forecasts and Trend Analysis, by Treatment, 2018 to 2030 (USD Billion)
  • 5.4. Gene Therapy
    • 5.4.1. Gene Therapy Market, 2018 - 2030 (USD Billion)
  • 5.5. Drug
    • 5.5.1. Drug Market, 2018 - 2030 (USD Billion)
    • 5.5.2. Spinraza
      • 5.5.2.1. Spinraza Market, 2018 - 2030 (USD Billion)
    • 5.5.3. Zolgensma (AVXS-101)
      • 5.5.3.1. Zolgensma (AVXS-101) Market, 2018 - 2030 (USD Billion)
    • 5.5.4. Evrysdi
      • 5.5.4.1. Evrysdi Market, 2018 - 2030 (USD Billion)
    • 5.5.5. Others
      • 5.5.5.1. Others Market, 2018 - 2030 (USD Billion)

Chapter 6. Spinal Muscular Atrophy Treatment Market: Route of Administration Business Analysis

  • 6.1. Route of Administration Market Share, 2024 & 2030
  • 6.2. Route of Administration Segment Dashboard
  • 6.3. Market Size & Forecasts and Trend Analysis, by Route of Administration, 2018 to 2030 (USD Billion)
  • 6.4. Oral
    • 6.4.1. Oral Market, 2018 - 2030 (USD Billion)
  • 6.5. Injection
    • 6.5.1. Injection Market, 2018 - 2030 (USD Billion)

Chapter 7. Spinal Muscular Atrophy Treatment Market: Regional Estimates & Trend Analysis

  • 7.1. Regional Market Share Analysis, 2024 & 2030
  • 7.2. Regional Market Dashboard
  • 7.3. Market Size & Forecasts Trend Analysis, 2018 to 2030:
  • 7.4. North America
    • 7.4.1. North America Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
    • 7.4.2. U.S.
      • 7.4.2.1. Key Country Dynamics
      • 7.4.2.2. Regulatory Framework
      • 7.4.2.3. Competitive Insights
      • 7.4.2.4. U.S. Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.4.3. Canada
      • 7.4.3.1. Key Country Dynamics
      • 7.4.3.2. Regulatory Framework
      • 7.4.3.3. Competitive Insights
      • 7.4.3.4. Canada Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.4.4. Mexico
      • 7.4.4.1. Key Country Dynamics
      • 7.4.4.2. Regulatory Framework
      • 7.4.4.3. Competitive Insights
      • 7.4.4.4. Mexico Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5. Europe
    • 7.5.1. Europe Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
    • 7.5.2. UK
      • 7.5.2.1. Key Country Dynamics
      • 7.5.2.2. Regulatory Framework
      • 7.5.2.3. Competitive Insights
      • 7.5.2.4. UK Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.3. Germany
      • 7.5.3.1. Key Country Dynamics
      • 7.5.3.2. Regulatory Framework
      • 7.5.3.3. Competitive Insights
      • 7.5.3.4. Germany Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.4. France
      • 7.5.4.1. Key Country Dynamics
      • 7.5.4.2. Regulatory Framework
      • 7.5.4.3. Competitive Insights
      • 7.5.4.4. France Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.5. Italy
      • 7.5.5.1. Key Country Dynamics
      • 7.5.5.2. Regulatory Framework
      • 7.5.5.3. Competitive Insights
      • 7.5.5.4. Italy Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.6. Spain
      • 7.5.6.1. Key Country Dynamics
      • 7.5.6.2. Regulatory Framework
      • 7.5.6.3. Competitive Insights
      • 7.5.6.4. Spain Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.7. Norway
      • 7.5.7.1. Key Country Dynamics
      • 7.5.7.2. Regulatory Framework
      • 7.5.7.3. Competitive Insights
      • 7.5.7.4. Norway Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.8. Denmark
      • 7.5.8.1. Key Country Dynamics
      • 7.5.8.2. Regulatory Framework
      • 7.5.8.3. Competitive Insights
      • 7.5.8.4. Denmark Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.5.9. Sweden
      • 7.5.9.1. Key Country Dynamics
      • 7.5.9.2. Regulatory Framework
      • 7.5.9.3. Competitive Insights
      • 7.5.9.4. Sweden Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.6. Asia Pacific
    • 7.6.1. Asia Pacific Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.2. Japan
      • 7.6.2.1. Key Country Dynamics
      • 7.6.2.2. Regulatory Framework
      • 7.6.2.3. Competitive Insights
      • 7.6.2.4. Japan Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.3. China
      • 7.6.3.1. Key Country Dynamics
      • 7.6.3.2. Regulatory Framework
      • 7.6.3.3. Competitive Insights
      • 7.6.3.4. China Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.4. India
      • 7.6.4.1. Key Country Dynamics
      • 7.6.4.2. Regulatory Framework
      • 7.6.4.3. Competitive Insights
      • 7.6.4.4. India Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.5. South Korea
      • 7.6.5.1. Key Country Dynamics
      • 7.6.5.2. Regulatory Framework
      • 7.6.5.3. Competitive Insights
      • 7.6.5.4. South Korea Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.6. Australia
      • 7.6.6.1. Key Country Dynamics
      • 7.6.6.2. Regulatory Framework
      • 7.6.6.3. Competitive Insights
      • 7.6.6.4. Australia Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.6.7. Thailand
      • 7.6.7.1. Key Country Dynamics
      • 7.6.7.2. Regulatory Framework
      • 7.6.7.3. Competitive Insights
      • 7.6.7.4. Thailand Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.7. Latin America
    • 7.7.1. Latin America Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
    • 7.7.2. Brazil
      • 7.7.2.1. Key Country Dynamics
      • 7.7.2.2. Regulatory Framework
      • 7.7.2.3. Competitive Insights
      • 7.7.2.4. Brazil Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.7.3. Argentina
      • 7.7.3.1. Key Country Dynamics
      • 7.7.3.2. Regulatory Framework
      • 7.7.3.3. Competitive Insights
      • 7.7.3.4. Argentina Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8. MEA
    • 7.8.1. MEA Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
    • 7.8.2. South Africa
      • 7.8.2.1. Key Country Dynamics
      • 7.8.2.2. Regulatory Framework
      • 7.8.2.3. Competitive Insights
      • 7.8.2.4. South Africa Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.8.3. Saudi Arabia
      • 7.8.3.1. Key Country Dynamics
      • 7.8.3.2. Regulatory Framework
      • 7.8.3.3. Competitive Insights
      • 7.8.3.4. Saudi Arabia Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.8.4. UAE
      • 7.8.4.1. Key Country Dynamics
      • 7.8.4.2. Regulatory Framework
      • 7.8.4.3. Competitive Insights
      • 7.8.4.4. UAE Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
    • 7.8.5. Kuwait
      • 7.8.5.1. Key Country Dynamics
      • 7.8.5.2. Regulatory Framework
      • 7.8.5.3. Competitive Insights
      • 7.8.5.4. Kuwait Spinal Muscular Atrophy Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)

Chapter 8. Competitive Landscape

  • 8.1. Participant Overview
  • 8.2. Company Market Position Analysis
  • 8.3. Company Categorization
  • 8.4. Strategy Mapping
  • 8.5. Company Profiles/Listing
    • 8.5.1. Biogen
      • 8.5.1.1. Overview
      • 8.5.1.2. Financial Performance
      • 8.5.1.3. Product Benchmarking
      • 8.5.1.4. Strategic Initiatives
    • 8.5.2. Novartis AG
      • 8.5.2.1. Overview
      • 8.5.2.2. Financial Performance
      • 8.5.2.3. Product Benchmarking
      • 8.5.2.4. Strategic Initiatives
    • 8.5.3. Pfizer Inc.
      • 8.5.3.1. Overview
      • 8.5.3.2. Financial Performance
      • 8.5.3.3. Product Benchmarking
      • 8.5.3.4. Strategic Initiatives
    • 8.5.4. Ionis Pharmaceuticals
      • 8.5.4.1. Overview
      • 8.5.4.2. Financial Performance
      • 8.5.4.3. Product Benchmarking
      • 8.5.4.4. Strategic Initiatives
    • 8.5.5. Biohaven, Ltd.
      • 8.5.5.1. Overview
      • 8.5.5.2. Financial Performance
      • 8.5.5.3. Product Benchmarking
      • 8.5.5.4. Strategic Initiatives
    • 8.5.6. F. Hoffmann-La Roche Ltd
      • 8.5.6.1. Overview
      • 8.5.6.2. Financial Performance
      • 8.5.6.3. Product Benchmarking
      • 8.5.6.4. Strategic Initiatives
    • 8.5.7. Cytokinetics
      • 8.5.7.1. Overview
      • 8.5.7.2. Financial Performance
      • 8.5.7.3. Product Benchmarking
      • 8.5.7.4. Strategic Initiatives
    • 8.5.8. Scholar Rock, Inc.
      • 8.5.8.1. Overview
      • 8.5.8.2. Financial Performance
      • 8.5.8.3. Product Benchmarking
      • 8.5.8.4. Strategic Initiatives
    • 8.5.9. PTC Therapeutics
      • 8.5.9.1. Overview
      • 8.5.9.2. Financial Performance
      • 8.5.9.3. Product Benchmarking
      • 8.5.9.4. Strategic Initiatives
    • 8.5.10. NMD PHARMA A/S
      • 8.5.10.1. Overview
      • 8.5.10.2. Financial Performance
      • 8.5.10.3. Product Benchmarking
      • 8.5.10.4. Strategic Initiatives

List of Tables

  • Table 1 List of secondary sources
  • Table 2 List of abbreviations
  • Table 3 Global spinal muscular atrophy treatment market, by region, 2018 - 2030 (USD Billion)
  • Table 4 Global spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 5 Global spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 6 Global spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 7 North America spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
  • Table 8 North America spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 9 North America spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 10 North America spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 11 U.S. spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 12 U.S. spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 13 U.S. spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 14 Canada spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 15 Canada spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 16 Canada spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 17 Mexico spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 18 Mexico spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 19 Mexico spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 20 Europe spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
  • Table 21 Europe spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 22 Europe spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 23 Europe spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 24 UK spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 25 UK spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 26 UK spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 27 Germany spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 28 Germany spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 29 Germany spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 30 France spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 31 France spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 32 France spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 33 Italy spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 34 Italy spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 35 Italy spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 36 Spain spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 37 Spain spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 38 Spain spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 39 Norway spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 40 Norway spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 41 Norway spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 42 Denmark spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 43 Denmark spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 44 Denmark spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 45 Sweden spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 46 Sweden spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 47 Sweden spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 48 Asia Pacific spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
  • Table 49 Asia Pacific spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 50 Asia Pacific spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 51 Asia Pacific spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 52 Japan spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 53 Japan spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 54 Japan spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 55 China spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 56 China spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 57 China spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 58 India spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 59 India spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 60 India spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 61 Australia spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 62 Australia spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 63 Australia spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 64 South Korea spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 65 South Korea spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 66 South Korea spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 67 Thailand spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 68 Thailand spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 69 Thailand spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 70 Latin America spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
  • Table 71 Latin America spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 72 Latin America spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion
  • Table 73 Latin America spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion
  • Table 74 Brazil spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 75 Brazil spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 76 Brazil spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 77 Argentina spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 78 Argentina spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 79 Argentina spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 80 Middle East & Africa spinal muscular atrophy treatment market, by country, 2018 - 2030 (USD Billion)
  • Table 81 Middle East & Africa spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 82 Middle East & Africa spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 83 Middle East & Africa spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 84 South Africa spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 85 South Africa spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 86 South Africa spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 87 Saudi Arabia spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 88 Saudi Arabia spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 89 Saudi Arabia spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 90 UAE spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 91 UAE spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 92 UAE spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)
  • Table 93 Kuwait spinal muscular atrophy treatment market, by type, 2018 - 2030 (USD Billion)
  • Table 94 Kuwait spinal muscular atrophy treatment market, by treatment, 2018 - 2030 (USD Billion)
  • Table 95 Kuwait spinal muscular atrophy treatment market, by route of administration, 2018 - 2030 (USD Billion)

List of Figures

  • Fig. 1 Spinal muscular atrophy treatment market segmentation
  • Fig. 2 Market research process
  • Fig. 3 Data triangulation techniques
  • Fig. 4 Primary research pattern
  • Fig. 5 Market research approaches
  • Fig. 6 Value-chain-based sizing & forecasting
  • Fig. 7 Market formulation & validation
  • Fig. 8 Market snapshot
  • Fig. 9 Type outlook (USD Billion)
  • Fig. 10 Treatment outlook (USD Billion)
  • Fig. 11 Route of administration outlook (USD Billion)
  • Fig. 12 Competitive landscape
  • Fig. 13 Spinal muscular atrophy treatment market dynamics
  • Fig. 14 Spinal muscular atrophy treatment market: Porter's five forces analysis
  • Fig. 15 Spinal muscular atrophy treatment market: PESTLE analysis
  • Fig. 16 Spinal muscular atrophy treatment market: Type segment dashboard
  • Fig. 17 Spinal muscular atrophy treatment market: Type market share analysis, 2024 & 2030
  • Fig. 18 Type 1 market, 2018 - 2030 (USD Billion)
  • Fig. 19 Type 2 market, 2018 - 2030 (USD Billion)
  • Fig. 20 Type 3 market, 2018 - 2030 (USD Billion)
  • Fig. 21 Type 4 market, 2018 - 2030 (USD Billion)
  • Fig. 22 Spinal muscular atrophy treatment market: Treatment segment dashboard
  • Fig. 23 Spinal muscular atrophy treatment market: Treatment market share analysis, 2024 & 2030
  • Fig. 24 Gene therapy market, 2018 - 2030 (USD Billion)
  • Fig. 25 Drug market, 2018 - 2030 (USD Billion)
  • Fig. 26 Spinraza market, 2018 - 2030 (USD Billion)
  • Fig. 27 Zolgensma (AVXS-101) market, 2018 - 2030 (USD Billion)
  • Fig. 28 Evrysdi market, 2018 - 2030 (USD Billion)
  • Fig. 29 Others market, 2018 - 2030 (USD Billion)
  • Fig. 30 Spinal muscular atrophy treatment market: Route of administration segment dashboard
  • Fig. 31 Spinal muscular atrophy treatment market: Route of administration market share analysis, 2024 & 2030
  • Fig. 32 Oral market, 2018 - 2030 (USD Billion)
  • Fig. 33 Injection market, 2018 - 2030 (USD Billion)
  • Fig. 34 Spinal muscular atrophy treatment market revenue, by region
  • Fig. 35 Regional marketplace: Key takeaways
  • Fig. 36 North America spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 37 U.S. country dynamics
  • Fig. 38 U.S. spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 39 Canada country dynamics
  • Fig. 40 Canada spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 41 Mexico country dynamics
  • Fig. 42 Mexico spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 43 Europe spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 44 UK country dynamics
  • Fig. 45 UK spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 46 Germany country dynamics
  • Fig. 47 Germany spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 48 France country dynamics
  • Fig. 49 France spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 50 Italy country dynamics
  • Fig. 51 Italy spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 52 Spain country dynamics
  • Fig. 53 Spain spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 54 Norway country dynamics
  • Fig. 55 Norway spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 56 Denmark country dynamics
  • Fig. 57 Denmark spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion
  • Fig. 58 Sweden country dynamics
  • Fig. 59 Sweden spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 60 Asia Pacific spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 61 Japan country dynamics
  • Fig. 62 Japan spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 63 China country dynamics
  • Fig. 64 China spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 65 India country dynamics
  • Fig. 66 India spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 67 South Korea country dynamics
  • Fig. 68 South Korea spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 69 Australia country dynamics
  • Fig. 70 Australia spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 71 Thailand country dynamics
  • Fig. 72 Thailand spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 73 Latin America spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 74 Brazil country dynamics
  • Fig. 75 Brazil spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 76 Argentina country dynamics
  • Fig. 77 Argentina spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 78 MEA spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 79 South Africa country dynamics
  • Fig. 80 South Africa spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 81 Saudi Arabia country dynamics
  • Fig. 82 Saudi Arabia spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 83 UAE country dynamics
  • Fig. 84 UAE spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 85 Kuwait country dynamics
  • Fig. 86 Kuwait spinal muscular atrophy treatment market, 2018 - 2030 (USD Billion)
  • Fig. 87 Company categorization
  • Fig. 88 Company market position analysis
  • Fig. 89 Strategic framework