查看購物車
  • Simplified Chinese
  • English
  • Japanese
  • Korean
封面
市場調查報告書
商品編碼
1632615

全球脊髓性肌肉萎縮症治療市場：市場規模、佔有率和趨勢分析（按類型、治療方法、給藥途徑、地區和細分，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個工作天內

價格

脊髓性肌肉萎縮症治療市場的成長與趨勢：

根據Grand View Research Inc.的最新研究，全球脊髓性肌肉萎縮症治療市場規模預計到2030年將達到130.9億美元，2025年至2030年複合年成長率為18.0%。

這一成長主要得益於產品核准和上市數量的增加。例如，2020年8月，基因泰克開發的Evrysdi（risdiplam）獲得食品藥物管理局核准，用於治療2個月及以上的脊髓性肌肉萎縮症（SMA）兒科患者。

此外，2020年5月，諾華公司SMA候選藥物Zolgensma（onasemnogene abeparvovec）獲得歐盟委員會（EC）有條件核准，用於治療SMA患者。這是唯一在歐洲核准的基因治療藥物。 2021年3月，PTC Therapeutics獲得EMA授予的Evrysdi（risdiplam）上市核准，用於治療1型、2型和3型脊髓性肌肉萎縮症患者。因此，預計產品核可數量的增加將推動脊髓性肌肉萎縮治療市場的發展。

諾華公司透過其全球管理存取計畫 (MAP) 免費提供藥物，為 SMA 患者提供支援。該計劃向全球符合條件的 SMA 患者免費提供 100 劑 Zolgenma，包括北美洲和南美洲、亞洲、澳洲、歐洲和非洲。因此，Zolgensma 此類支援計畫的存在也有助於加強促銷力度並創造收益。

全球新生兒篩檢和便利的診斷工具取得應成為常規方法的一部分。這將確保早期診斷並及時治療。例如，2021 年 7 月，北卡羅來納州教堂山北卡羅來納大學醫學中心的嬰兒夏洛特透過 EarlyCheck新生兒篩檢計畫被診斷出患有脊髓性肌肉萎縮症 (SMA)，並接受了具有突破性的基因治療。此類篩檢計畫有望促進該疾病的早期診斷和治療。

此外，製藥公司正與研究機構進行多項大規模合作，開發治療脊髓性肌肉萎縮症患者的新治療方法。例如，2021年3月，PTC Therapeutics, Inc.與脊髓性肌肉萎縮症基金會達成研究合作，以推進科學研究和開發治療脊髓性肌肉萎縮症患者的新療法。

然而，治療相關的高成本以及參與企業數量少可能會在預測期內抑制市場的成長。孤兒藥研發高成本是產品價格上漲的主要因素。例如，最常用的藥物 Spinraza 每次注射的費用約為 125,000 美元，每年的費用為 750,000 美元。此外，Zolgensma (AVXS-101) 每次治療的費用為 210 萬美元。

脊髓性肌肉萎縮症治療市場：分析概述

  • 根據類型，1 型脊髓性肌肉萎縮症在 2024 年佔據了最大的市場佔有率，這歸因於 1 型脊髓性肌肉萎縮症患者的高盛行率和治療產品的廣泛可用性。
  • 基於治療方法，由於市場滲透率的不斷提高，預計基因治療領域在預測期內將成長最快。 Zolgensma 是唯一在 37 個國家核准治療 SMA 患者的基因療法。
  • 基於藥物，由於優惠的保險報銷政策和產品知名度的提高，Spinraza 細分市場將在 2024 年佔據市場主導地位。 SPINRAZA 目前在義大利、挪威和荷蘭三個歐洲國家已全額報銷。
  • 根據給藥途徑，注射劑在 2024 年佔據了市場主導地位，因為透過這種給藥途徑向 SMA 患者注射藥物具有很高的安全性、有效性和耐受性。
  • 預計預測期內亞太地區將成長最快。該地區的成長得益於新產品的進入。例如，2021年7月，羅氏公司在印度推出了Evrysdi（risdiplam），用於治療脊髓性肌肉萎縮症患者。

目錄

第1章 分析方法及範圍

第 2 章執行摘要

3. 脊髓性肌肉萎縮症治療市場：促進因素、趨勢與範圍

  • 市場聯動展望
  • 市場動態
    • 市場促進因素分析
    • 市場限制分析
  • 商業環境分析
    • 產業分析：波特五力分析
    • PESTEL 分析

第 4 章脊髓性肌肉萎縮症治療市場：按類型分類的商業分析

  • 按類型分類的市場佔有率（2024 年和 2030 年）
  • 按類型分類的儀表板
  • 按類型分類的市場規模預測及趨勢分析（2018-2030）
  • 類型 1
  • 類型 2
  • 類型 3
  • 類型 4

第 5 章脊髓性肌肉萎縮症治療市場：按治療方法進行的商業分析

  • 治療方法的市場佔有率（2024 年和 2030 年）
  • 治療方法儀表板
  • 市場規模預測及治療方法趨勢分析（2018-2030）
  • 基因治療
  • 藥物

6. 脊髓性肌肉萎縮症治療市場：依給藥途徑分類的業務分析

  • 按管理途徑分類的市場佔有率（2024 年和 2030 年）
  • 管理路線儀表板
  • 依管理途徑分類的市場規模預測及趨勢分析（2018-2030 年）
  • 口服
  • 注射

7. 脊髓性肌肉萎縮症治療市場：各地區業務分析

  • 按地區分類的市場佔有率分析（2024 年和 2030 年）
  • 市場儀表板：按地區
  • 市場規模預測及趨勢分析（2018-2030年）
  • 北美洲
    • 按國家/地區，2018 年至 2030 年
    • 美國
    • 加拿大
    • 墨西哥
  • 歐洲
    • 按國家/地區，2018 年至 2030 年
    • 英國
    • 德國
    • 法國
    • 義大利
    • 西班牙
    • 挪威
    • 丹麥
    • 瑞典
  • 亞太地區
    • 日本
    • 中國
    • 印度
    • 韓國
    • 澳洲
    • 泰國
  • 拉丁美洲
    • 按國家/地區，2018 年至 2030 年
    • 巴西
    • 阿根廷
  • 中東和非洲
    • 按國家/地區，2018 年至 2030 年
    • 南非
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國
    • 科威特

第8章 競爭格局

  • 市場參與企業概況
  • 公司市場地位分析
  • 公司分類
  • 戰略地圖
  • 公司簡介/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