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市场调查报告书
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1425120

全球骨骼发育不良市场：预测（2024-2029）

Skeletal Dysplasia Market - Forecasts from 2024 to 2029
出版日期: | 出版商: Knowledge Sourcing Intelligence | 英文 149 Pages | 商品交期: 最快1-2个工作天内

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简介目录

预测期内，全球骨骼发育不良市场规模预计将以 3.69% 的复合年增长率成长。

骨软骨发育不良，又称骨骼发育不良（SD），是一组严重影响骨骼的罕见且多样化的疾病。这些疾病由于软骨和骨骼发育不当而导致骨骼长度、型态和密度异常。儘管个别疾病很少见，但 SD 的平均发生率至少为每 5,000 名活产儿中就有 1 人发病，已发现 461 种疾病。不同的临床表现，包括生长、骨型态和骨骼密度，证明了发病机制的复杂性。

软骨发育不全盛行率增加

软骨发育不全是一种遗传性疾病，会导致生长不对称和身材矮小。患有软骨发育不全的成年人通常身高不到 4 英尺。软骨发育不全是一种遗传性疾病，其中称为纤维母细胞细胞生长因子受体 3 的生长调节基因过度活跃，从而阻止正常的骨骼发育。根据美国国家科学图书馆的数据，到 2023 年，超过 90% 的身材过小（俗称侏儒症）病例将由这种类型的发育不良引起。

政府努力治疗罕见疾病

罕见疾病在全球范围内流行，导致骨骼发育不良市场的成长。因此，世界各国政府都在关注各种措施。例如，根据2021年国家罕见疾病政策，卫生和家庭福利部创建了一个数位入口网站，用于罕见疾病患者的群众集资和自愿捐赠。作为 ICMR 发起的国家登记的一部分，正在收集有关罕见疾病和其他遗传疾病的流行病学资讯。收集病例多达4001例，包括储存障碍、听小骨原发性代谢缺陷、原发性免疫力缺乏、骨骼神经肌肉、神经肌肉疾病、血红蛋白疾病和出血性疾病。

骨骼发育不良认识计划

许多公司和政府机构都专注于提高认识计划，进一步促进骨骼发育不良市场的成长。例如，2023年10月，约翰霍普金斯大学医学院的专家为侏儒症和其他骨病患者进行了历史最长的门诊。该诊所现称为凯瑟琳和艾伦·格林伯格骨骼发育不良中心，成立于 20 世纪 50 年代末，旨在提供资源和医疗援助。我们也教育、培训和僱用医护人员，以改善侏儒症患者的生活。此外，非营利性的欧洲骨骼发育不良网路（ESDN）也提供了全面的骨骼发育不良研究和诊断网路。 ESDN 的两个目标是开发有效的方法来诊断成骨不全症，并了解哪些细胞、分子和遗传因素与成骨不全症有关。

骨骼发育不良市场的技术进步

以更简单、更快速、更经济且用户友好的方式进行即时监测的生物感测器的研究和开发正在进行中，以推动骨骼发育不良市场的发展。大多数骨生物感测器只能检测一种类型的骨健康指标。由于单一骨生物标记的检测不足以准确、快速地诊断骨骼疾病，因此许多生物标记的检测正在建立。为了解决这些问题，需要能够同时检测多种分析物的多重检测技术。据说多重检测更加灵敏并且需要更小的样本大小。为了解决日益严重的骨骼健康问题，骨骼发育不良产业正在寻求先进的生物感测器，可以使用无标定检测方法检测多种指标。

亚太地区骨骼发育不良市场预计将稳定成长。

预计亚太地区骨骼发育不良市场将受到骨骼发育不良行业主要企业的存在以及该地区各种倡议的增长的推动。例如，上海市骨病临床研究中心（SCRCB）是中国为数不多的专门治疗骨质疏鬆症和罕见骨骼疾病的中心之一。它也是为数不多的研究目的免费对骨骼疾病受试者进行基因分析的中心之一。因此，许多疑似病例前往该中心寻求明确诊断。此外，印度政府也为身材矮小、骨骼发育不良、轮椅使用者等提供不同高度的服务台。亚太国家的这些努力正在推动该地区的骨骼发育不良市场。

骨骼发育不良的治疗

  • 圆头畸形：每次与医疗保健提供者会面时都会测量患者的头围，并根据软骨发育不全特定的监测标准进行绘製。
  • 椎间孔狭窄：美国小儿科会建议 FMS 患者先进行睡眠研究（多导睡眠图），如果呈阳性，则进行电脑断层扫描或 MRI 检查颈椎区域的压迫性脊髓型颈椎病变。
  • 胸腰椎后凸 (TLK)：TLK 通常发生在新生儿中，90% 的受影响儿童在 18 个月大时会自行恢復，因为他们的行走能力随着身体躯干肌肉的发展而提高。

骨骼发育不良临床试验

  • 2023 年 5 月，Nemours Children's Health 将作为美国国立卫生研究院 (FNIH) 加速药物合作伙伴关係®定制基因治疗联盟基金会的成员，对 Morquio A 综合征进行突破性的基因治疗临床试验。
  • 2023 年 3 月，BridgeBio Pharma, Inc. 报告了 PROPEL2 的积极结果，这是一项针对软骨发育不全儿童的临床实验药物 infigratinib 的 2 期研究。 BridgeBio Pharma, Inc 是一家商业阶段的生物製药公司，专注于遗传疾病和癌症领域。这些结果显示该药物具有一流的疗效和较低的副作用风险。

目录

第一章简介

  • 市场概况
  • 市场定义
  • 调查范围
  • 市场区隔
  • 货币
  • 先决条件
  • 基准年和预测年时间表

第二章调查方法

  • 调查资料
  • 先决条件

第三章执行摘要

  • 研究亮点

第四章市场动态

  • 市场驱动因素
  • 市场限制因素
  • 波特五力分析
  • 产业价值链分析

第五章骨骼发育不良市场：依类型

  • 介绍
  • 莫尔基奥A症候群
  • X连锁低血磷症
  • 低磷酸酯酶症
  • 其他的

第六章骨骼发育不良市场：依治疗分类

  • 介绍
  • 酵素替代疗法
  • 人类单株抗体
  • 其他的

第七章骨骼发育不良市场：依通路

  • 介绍
  • 医院药房
  • 药局和专业药局
  • 其他的

第八章骨骼发育不良市场：依地区

  • 介绍
  • 北美洲
    • 美国
    • 加拿大
    • 墨西哥
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他的
  • 欧洲
    • 英国
    • 德国
    • 法国
    • 西班牙
    • 其他的
  • 中东/非洲
    • 沙乌地阿拉伯
    • 阿拉伯聯合大公国
    • 以色列
    • 其他的
  • 亚太地区
    • 日本
    • 中国
    • 印度
    • 韩国
    • 印尼
    • 泰国
    • 其他的

第九章竞争环境及分析

  • 主要企业及策略分析
  • 市场占有率分析
  • 併购/协议/合作
  • 供应商竞争力矩阵

第十章 公司简介

  • Johns Hopkins Medicine
  • Cedars-Sinai
  • Igenomix India
  • Blueprint Genetics
  • Invitae
  • GeneDx
  • HealthPoint
  • Cegat
  • Varsomics
简介目录
Product Code: KSI061616215

The skeletal dysplasia market is estimated to grow at a CAGR of 3.69% during the forecast period.

Osteochondrodysplasia, another name for skeletal dysplasias (SD), is a set of uncommon, diverse illnesses that significantly affect the skeleton. These disorders cause aberrant bone length, form, and density due to improper cartilage and bone development. Despite being uncommon individually, SD has an average incidence of at least 1 per 5,000 live births, with 461 entities having been identified. The variety of clinical symptoms, including growth, bone form, and bone density, illustrates the complexity of the etiopathogenetic process.

Growing prevalence of achondroplasia

Achondroplasia is a genetic disorder that results in asymmetrical development and very small height. Adults with achondroplasia often stand four feet or less tall. Achondroplasia is a hereditary disorder that hinders normal bone development by making a growth-regulatory gene called fibroblast growth factor receptor 3 overactive. According to the National Library of Science, in 2023, more than 90% of instances of disproportionately small height, popularly known as dwarfism, are caused by this kind of dysplasia.

Government initiatives for the treatment of rare diseases

Rare diseases have been prevailing worldwide leading to growth in the skeletal dysplasia market. Owing to this, the government around the world is focusing on various initiatives. For instance, in line with the National Policy for Rare Diseases, 2021, the Department of Health and Family Welfare has created a digital portal for crowdfinancing and voluntary donations for patients with rare diseases. Epidemiological information is being gathered for rare illnesses and other hereditary disorders as part of a National Registry that the ICMR has started. Up to 4001 instances of uncommon diseases, including storage disorders, small meta bone inborn metabolic errors, primary immune deficiency disorders, skeletal dysplasia, neuromuscular disorders, hemoglobinopathies, and bleeding disorders, have been gathered.

Awareness programs for skeletal dysplasia

Many companies and government organizations are focusing on awareness programs further boosting the skeletal dysplasia market growth. For instance, in October 2023, the longest-running clinic for patients with dwarfism and other bone disorders was conducted by specialists from Johns Hopkins Medicine. The clinic, presently known as the Kathryn and Alan C. Greenberg Centre for Skeletal Dysplasia, was founded in the late 1950s and offers resources and medical assistance. To better the lives of persons with dwarfism, it also educates, trains, and employs healthcare workers. Further, a comprehensive research and diagnostic network for skeletal dysplasias is offered by the non-profit European Skeletal Dysplasia Network (ESDN). The ESDN's two goals are to create efficient methods for the diagnosis of bone dysplasias and to comprehend what cellular, molecular, and genetic elements contribute to bone dysplasias.

Technological advancements in the skeletal dysplasia market

Research and development of biosensors for real-time monitoring with easier, faster, more affordable, and user-friendly methods are used which fuels the skeletal dysplasia market. The majority of bone biosensors only find one type of bone health indicator. The detection of many biomarkers is being established since the detection of a single bone biomarker is insufficient for the precise and prompt diagnosis of bone disorders. To solve these issues, multiplex detection techniques that can detect many analytes concurrently are needed. Multiplex assays are said to be very sensitive and call for a smaller sample size. To address the growing issues with bone health, sophisticated biosensors that can detect various indicators using a label-free detection approach are required in the skeletal dysplasia industry.

Skeletal dysplasia market in the Asia Pacific region is anticipated to grow steadily.

The skeletal dysplasia market in the Asia Pacific is anticipated to be fueled by the presence of major skeletal dysplasia industry players and growth in various initiatives in the region. For instance, the Shanghai Clinical Research Centre of Bone Disease (SCRCB) is one of the few established centers in China that focuses on the treatment of osteoporosis and rare skeletal disorders. It is also one of the few centers that offers free genetic analysis for skeletal disorder subjects for research purposes. As a result, many suspected cases come to this center for a confirmed diagnosis. Additionally, the Indian government provides service desks at various heights for those with short stature, skeletal dysplasia, and wheelchair users, among others. These initiatives by various Asia Pacific countries are fueling the skeletal dysplasia market in the region.

Skeletal Dysplasia Treatment

  • Marcocephaly, every time a patient interacts with a healthcare professional, head circumference measurements are taken and plotted on criteria for monitoring specific to achondroplasia.
  • Foramen Magnum Stenosis, the American Academy of Paediatrics advises getting a sleep study (polysomnography) for FMS patients first, and if it is positive, getting a CT scan or MRI to check for compressive myelopathy in the cervicomedullary region. If either examination yields aberrant results, immediate neurosurgical assessment are scheduled for potential surgical intervention.
  • Thoracolumbar Kyphosis (TLK), TLK frequently manifests in the newborn and, in 90% of afflicted children, resolves spontaneously by the age of 18 months, coinciding with the child's improvement in their ability to walk as their trunk muscles develop.

Clinical trials for skeletal dysplasia

  • In May 2023, Nemours Children's Health was selected to carry out a ground-breaking gene therapy clinical study for Morquio A syndrome as a member of the Foundation for the National Institutes of Health (FNIH) accelerating medicines partnership® Bespoke Gene Therapy Consortium.
  • In March 2023, Positive results from PROPEL2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were reported by BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company specializing in genetic diseases and cancers. These results suggest potential best-in-class efficacy and a low risk of side effects.

Market Key Developments

  • In August 2023, the U.S. Food and Drug Administration (FDA) designated Tyra Biosciences, Inc.'s, lead precision medicine program, TYRA-300, as an orphan drug for the treatment of achondroplasia. Tyra Biosciences, Inc. is a clinical-stage biotechnology company dedicated to developing next-generation precision medicines that target significant unmet medical needs in fibroblast growth factor receptor (FGFR) biology.
  • In November 2021, BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval to VOXZOGOTM (vosoritide for injection), which is intended to promote linear growth in children with achondroplasia who are five years of age or older and have open epiphyses (growth plates).

Segmentation:

By Type

  • Morquio A Syndrome
  • X-linked Hypophosphatemia
  • Hypophosphatasia
  • Others

By Treatment

  • Enzyme Replacement Therapy
  • Human Monoclonal Antibody
  • Others

By Distribution Channel

  • Hospital Pharmacies
  • Drug Stores and Specialty Pharmacies
  • Others

By Geography

  • North America
  • United States
  • Canada
  • Mexico
  • South America
  • Brazil
  • Argentina
  • Others
  • Europe
  • United Kingdom
  • Germany
  • France
  • Spain
  • Others
  • Middle East and Africa
  • Saudi Arabia
  • UAE
  • Israel
  • Others
  • Asia Pacific
  • Japan
  • China
  • India
  • South Korea
  • Indonesia
  • Thailand
  • Others

TABLE OF CONTENTS

1. INTRODUCTION

  • 1.1. Market Overview
  • 1.2. Market Definition
  • 1.3. Scope of the Study
  • 1.4. Market Segmentation
  • 1.5. Currency
  • 1.6. Assumptions
  • 1.7. Base, and Forecast Years Timeline

2. RESEARCH METHODOLOGY

  • 2.1. Research Data
  • 2.2. Assumptions

3. EXECUTIVE SUMMARY

  • 3.1. Research Highlights

4. MARKET DYNAMICS

  • 4.1. Market Drivers
  • 4.2. Market Restraints
  • 4.3. Porter's Five Force Analysis
    • 4.3.1. Bargaining Power of Suppliers
    • 4.3.2. Bargaining Power of Buyers
    • 4.3.3. Threat of New Entrants
    • 4.3.4. Threat of Substitutes
    • 4.3.5. Competitive Rivalry in the Industry
  • 4.4. Industry Value Chain Analysis

5. SKELETAL DYSPLASIA MARKET BY TYPE

  • 5.1. Introduction
  • 5.2. Morquio A Syndrome
  • 5.3. X-linked Hypophosphatemia
  • 5.4. Hypophosphatasia
  • 5.5. Others

6. SKELETAL DYSPLASIA MARKET BY TREATMENT

  • 6.1. Introduction
  • 6.2. Enzyme Replacement Therapy
  • 6.3. Human Monoclonal Antibody
  • 6.4. Others

7. SKELETAL DYSPLASIA MARKET BY DISTRIBUTION CHANNEL

  • 7.1. Introduction
  • 7.2. Hospital Pharmacies
  • 7.3. Drug Stores and Specialty Pharmacies
  • 7.4. Others

8. SKELETAL DYSPLASIA MARKET BY GEOGRAPHY

  • 8.1. Introduction
  • 8.2. North America
    • 8.2.1. United States
    • 8.2.2. Canada
    • 8.2.3. Mexico
  • 8.3. South America
    • 8.3.1. Brazil
    • 8.3.2. Argentina
    • 8.3.3. Others
  • 8.4. Europe
    • 8.4.1. United Kingdom
    • 8.4.2. Germany
    • 8.4.3. France
    • 8.4.4. Spain
    • 8.4.5. Others
  • 8.5. The Middle East and Africa
    • 8.5.1. Saudi Arabia
    • 8.5.2. UAE
    • 8.5.3. Israel
    • 8.5.4. Others
  • 8.6. Asia Pacific
    • 8.6.1. Japan
    • 8.6.2. China
    • 8.6.3. India
    • 8.6.4. South Korea
    • 8.6.5. Indonesia
    • 8.6.6. Thailand
    • 8.6.7. Others

9. COMPETITIVE ENVIRONMENT AND ANALYSIS

  • 9.1. Major Players and Strategy Analysis
  • 9.2. Market Share Analysis
  • 9.3. Mergers, Acquisitions, Agreements, and Collaborations
  • 9.4. Vendor Competitiveness Matrix

10. COMPANY PROFILES

  • 10.1. Johns Hopkins Medicine
  • 10.2. Cedars-Sinai
  • 10.3. Igenomix India
  • 10.4. Blueprint Genetics
  • 10.5. Invitae
  • 10.6. GeneDx
  • 10.7. HealthPoint
  • 10.8. Cegat
  • 10.9. Varsomics