脊髓性肌肉萎缩症治疗市场、机会、成长动力、产业趋势分析与预测，2024-2032

全球脊髓性肌肉萎缩症 (SMA) 治疗市场价值为 73 亿美元，预计 2024 年至 2032 年复合年增长率为 19.1%。主要驱动因素包括扩大新生儿筛检计划，从而实现早期诊断并提高治疗资格。早期诊断可以改善患者的治疗效果并增加对神经系统药物的需求。 Health Union, LLC 报告称，全球每 6,000 至 10,000 名新生儿中就有 1 人受 SMA 影响，其中 1 型 SMA 最为严重，占所有病例的一半以上。目前大约有 25,000 名美国人患有 SMA。诊断技术的进步以及医疗保健提供者和患者意识的提高预计将推动市场成长。

整个脊髓性肌肉萎缩症治疗产业根据类型、治疗类型、给药途径和地区进行分类。

全球 SMA 治疗市场按类型分为婴儿 SMA、Werdnig-Hoffmann 病、成人 SMA 和 Kugelberg-Welander 病。韦德尼格-霍夫曼病细分市场到2023 年以29 亿美元的销售额引领市场。及时治疗。这一趋势将推动市场成长。研究和开发以及降低治疗成本的製药合作扩大了患者的取得范围，进一步推动了市场成长。 SMA 1 型治疗的早期使用或同情使用计划也增强了市场动力。

按治疗类型划分，市场包括药物、支持性护理和脊椎手术。包括反义寡核苷酸、基因治疗和其他药物在内的药物细分市场，到2023年将占64.2%的份额。 SMN蛋白，改变了SMA治疗方法产量，拉动市场成长。注射剂和口服製剂的出现增加了治疗的可及性，满足了不同患者的需求和偏好，推动了市场的成长。

北美，尤其是美国，到 2023 年将占据全球 SMA 治疗市场 45.6% 的份额，预计将保持其主导地位。对罕见疾病的支持政策、先进的医疗设施和正在进行的临床试验是推动该地区市场成长的关键因素。此外，领先製药公司的存在以及研发资金的增加也进一步提振了市场。对脊髓性肌肉萎缩症认识的提高和早期诊断也有助于扩大治疗范围。

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • 提高婴儿和儿童脊髓性肌肉萎缩症的诊断率
    • 基因治疗的进展
    • 医疗保健提供者提高意识和筛检计划
  • 产业陷阱与挑战
    • 新疗法获得严格的监管批准
    • 治疗费用高
• 成长潜力分析
• 监管环境
• 未来市场趋势
• 管道分析
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司市占率分析
• 公司矩阵分析
• 主要市场参与者的竞争分析
• 竞争定位矩阵
• 战略仪表板

第 5 章：市场估计与预测：按类型，2021 - 2032

• 主要趋势
• 韦德尼格-霍夫曼病
• 婴儿SMA
• 库格伯格-韦兰德病
• 成人SMA

第 6 章：市场估计与预测：按治疗类型，2021 - 2032 年

• 主要趋势
• 药物
  • 基因治疗
  • 反义寡核苷酸
  • 其他药物
• 支持性护理
  • 物理治疗
  • 呼吸支持
  • 营养支持
• 脊椎手术

第 7 章：市场估计与预测：按管理途径，2021 - 2032 年

• 主要趋势
• 口服
• 注射用

第 8 章：市场估计与预测：按最终用途，2021 - 2032 年

• 主要趋势
• 医院
• 专科诊所
• 家庭护理设置
• 其他最终用户

第 9 章：市场估计与预测：按地区，2021 - 2032

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 荷兰
  • 欧洲其他地区
• 亚太地区
  • 日本
  • 中国
  • 印度
  • 澳洲
  • 韩国
  • 亚太地区其他地区
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 拉丁美洲其他地区
• 中东和非洲
  • 南非
  • 沙乌地阿拉伯
  • 阿联酋
  • 中东和非洲其他地区

第 10 章：公司简介

• American Physical Therapy Association
• Astellas Pharma
• Beijing Jinlan Gene Technology Co., Ltd.
• Biogen Inc.
• Boston's Children Hospital
• Children's Hospital of The King's Daughters
• F. Hoffmann-La Roche Ltd.
• Hanugen Therapeutics
• Nationwide Children's Hospital
• NMD Pharma A/S
• Novartis AG
• Pfizer, Inc.
• Sanofi (Genzyme Corporation)
• Scholar Rock, Inc

The Global Spinal Muscular Atrophy (SMA) Treatments Market was valued at USD 7.3 billion and is projected to grow at a CAGR of 19.1% from 2024-2032. Key drivers include the expansion of newborn screening programs, leading to earlier diagnoses and increased treatment eligibility. Early diagnosis enhances patient outcomes and boosts demand for neurological medications. Health Union, LLC reports that SMA affects 1 in every 6,000 to 10,000 newborns globally, with Type 1 SMA being the most severe and accounting for over half of all cases. Approximately 25,000 Americans currently live with SMA. Advancements in diagnostic technologies and increased awareness among healthcare providers and patients are anticipated to drive market growth.

The overall spinal muscular atrophy treatment industry is classified based on type, treatment type, route of administration, and region.

The global SMA treatment market is segmented by type into infant SMA, Werdnig-Hoffmann disease, adult SMA, and Kugelberg-Welander disease. The Werdnig-Hoffmann disease segment led the market with USD 2.9 billion in 2023. Mandatory newborn screenings have facilitated earlier detections, enabling prompt treatments that are most effective before significant motor neuron loss. This trend is set to drive market growth. Research and development, along with pharmaceutical collaborations to reduce treatment costs, have broadened patient access, further energizing market growth. Early access or compassionate use programs for SMA Type 1 treatment also bolster market momentum.

By treatment type, the market includes medication, supportive care, and spinal surgery. The medication segment, which includes antisense oligonucleotides, gene therapy, and other drugs, dominated with a 64.2% share in 2023. Innovative drugs like Spinraza (Nusinersen) and Evrysdi (Risdiplam) have transformed SMA treatment by targeting the genetic root and enhancing SMN protein production, driving market growth. The availability of both injectable and oral formulations has increased treatment accessibility, catering to diverse patient needs and preferences, thus fueling market growth.

North America, particularly the U.S., captured a significant 45.6% share of the global SMA treatment market in 2023 and is expected to maintain its dominance. Supportive policies for rare diseases, advanced healthcare facilities, and ongoing clinical trials are key factors driving market growth in the region. Additionally, the presence of leading pharmaceutical companies and increased funding for research and development are further bolstering the market. Rising awareness and early diagnosis of spinal muscular atrophy are also contributing to the expanding treatment landscape.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360°synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Increase in diagnosis rate of spinal muscular atrophy in infants and children
    • 3.2.1.2 Advancement in gene therapy
    • 3.2.1.3 Growing awareness and screening programs by healthcare providers
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory approval for new treatments
    • 3.2.2.2 High cost of treatment
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Future market trends
• 3.6 Pipeline analysis
• 3.7 Porter's analysis
• 3.8 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company market share analysis
• 4.3 Company matrix analysis
• 4.4 Competitive analysis of major market players
• 4.5 Competitive positioning matrix
• 4.6 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Werdnig-Hoffmann disease
• 5.3 Infant SMA
• 5.4 Kugelberg-Welander disease
• 5.5 Adult SMA

Chapter 6 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Medication
  • 6.2.1 Gene therapy
  • 6.2.2 Antisense oligonucleotide
  • 6.2.3 Other medications
• 6.3 Supportive care
  • 6.3.1 Physical therapy
  • 6.3.2 Respiratory support
  • 6.3.3 Nutritional support
• 6.4 Spinal surgery

Chapter 7 Market Estimates and Forecast, By Route of Administration, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 Oral
• 7.3 Parenteral

Chapter 8 Market Estimates and Forecast, By End-Use, 2021 - 2032 ($ Mn)

• 8.1 Key trends
• 8.2 Hospitals
• 8.3 Specialty clinics
• 8.4 Homecare settings
• 8.5 Other end-users

Chapter 9 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Mn)

• 9.1 Key trends
• 9.2 North America
  • 9.2.1 U.S.
  • 9.2.2 Canada
• 9.3 Europe
  • 9.3.1 Germany
  • 9.3.2 UK
  • 9.3.3 France
  • 9.3.4 Spain
  • 9.3.5 Italy
  • 9.3.6 Netherlands
  • 9.3.7 Rest of Europe
• 9.4 Asia Pacific
  • 9.4.1 Japan
  • 9.4.2 China
  • 9.4.3 India
  • 9.4.4 Australia
  • 9.4.5 South Korea
  • 9.4.6 Rest of Asia Pacific
• 9.5 Latin America
  • 9.5.1 Brazil
  • 9.5.2 Mexico
  • 9.5.3 Rest of Latin America
• 9.6 Middle East and Africa
  • 9.6.1 South Africa
  • 9.6.2 Saudi Arabia
  • 9.6.3 UAE
  • 9.6.4 Rest of Middle East and Africa

Chapter 10 Company Profiles

• 10.1 American Physical Therapy Association
• 10.2 Astellas Pharma
• 10.3 Beijing Jinlan Gene Technology Co., Ltd.
• 10.4 Biogen Inc.
• 10.5 Boston's Children Hospital
• 10.6 Children's Hospital of The King's Daughters
• 10.7 F. Hoffmann-La Roche Ltd.
• 10.8 Hanugen Therapeutics
• 10.9 Nationwide Children's Hospital
• 10.10 NMD Pharma A/S
• 10.11 Novartis AG
• 10.12 Pfizer, Inc.
• 10.13 Sanofi (Genzyme Corporation)
• 10.14 Scholar Rock, Inc