首頁 > 市場調查報告書 > 製藥

骨疾病

市場調查報告書

商品編碼

1425120

全球骨骼發育不良市場：預測（2024-2029）

Skeletal Dysplasia Market - Forecasts from 2024 to 2029

出版日期: 2024年01月24日 | 出版商:

Knowledge Sourcing Intelligence | 英文 149 Pages | 商品交期: 最快1-2個工作天內

價格

簡介目錄

預測期內，全球骨骼發育不良市場規模預計將以 3.69% 的複合年成長率成長。

骨軟骨發育不良，又稱骨骼發育不良（SD），是一組嚴重影響骨骼的罕見且多樣化的疾病。這些疾病由於軟骨和骨骼發育不當而導致骨骼長度、型態和密度異常。儘管個別疾病很少見，但 SD 的平均發生率至少為每 5,000 名活產兒中就有 1 人發病，已發現 461 種疾病。不同的臨床表現，包括生長、骨型態和骨骼密度，證明了發病機制的複雜性。

軟骨發育不全盛行率增加

軟骨發育不全是一種遺傳性疾病，會導致生長不對稱和身材矮小。患有軟骨發育不全的成年人通常身高不到 4 英尺。軟骨發育不全是一種遺傳性疾病，其中稱為纖維母細胞細胞生長因子受體 3 的生長調節基因過度活躍，從而阻止正常的骨骼發育。根據美國國家科學圖書館的數據，到 2023 年，超過 90% 的身材過小（俗稱侏儒症）病例將由這種類型的發育不良引起。

政府努力治療罕見疾病

罕見疾病在全球範圍內流行，導致骨骼發育不良市場的成長。因此，世界各國政府都在關注各種措施。例如，根據2021年國家罕見疾病政策，衛生和家庭福利部創建了一個數位入口網站，用於罕見疾病患者的群眾集資和自願捐贈。作為 ICMR 發起的國家登記的一部分，正在收集有關罕見疾病和其他遺傳疾病的流行病學資訊。收集病例多達4001例，包括儲存障礙、聽小骨原發性代謝缺陷、原發性免疫力缺乏、骨骼神經肌肉、神經肌肉疾病、血紅蛋白疾病和出血性疾病。

骨骼發育不良認知計劃

許多公司和政府機構都專注於提高認知計劃，進一步促進骨骼發育不良市場的成長。例如，2023年10月，約翰霍普金斯大學醫學院的專家為侏儒症和其他骨病患者進行了歷史最長的門診。該診所現稱為凱瑟琳和艾倫·格林伯格骨骼發育不良中心，成立於 20 世紀 50 年代末，旨在提供資源和醫療援助。我們也教育、培訓和僱用醫護人員，以改善侏儒症患者的生活。此外，非營利性的歐洲骨骼發育不良網路（ESDN）也提供了全面的骨骼發育不良研究和診斷網路。 ESDN 的兩個目標是開發有效的方法來診斷成骨不全症，並了解哪些細胞、分子和遺傳因素與成骨不全症有關。

骨骼發育不良市場的技術進步

以更簡單、更快速、更經濟且方便用戶使用的方式進行即時監測的生物感測器的研究和開發正在進行中，以推動骨骼發育不良市場的發展。大多數骨生物感測器只能檢測一種類型的骨健康指標。由於單一骨生物標記的檢測不足以準確、快速地診斷骨骼疾病，因此許多生物標記的檢測正在建立。為了解決這些問題，需要能夠同時檢測多種分析物的多重檢測技術。據說多重檢測更加靈敏並且需要更小的樣本大小。為了解決日益嚴重的骨骼健康問題，骨骼發育不良產業正在尋求先進的生物感測器，可以使用無標定檢測方法檢測多種指標。

亞太地區骨骼發育不良市場預計將穩定成長。

預計亞太地區骨骼發育不良市場將受到骨骼發育不良行業主要企業的存在以及該地區各種舉措的成長的推動。例如，上海市骨病臨床研究中心（SCRCB）是中國為數不多的專門治療骨質疏鬆症和罕見骨骼疾病的中心之一。它也是為數不多的研究目的免費對骨骼疾病受試者進行基因分析的中心之一。因此，許多疑似病例前往該中心尋求明確診斷。此外，印度政府也為身材矮小、骨骼發育不良、輪椅使用者等提供不同高度的服務台。亞太國家的這些努力正在推動該地區的骨骼發育不良市場。

骨骼發育不良的治療

圓頭畸形：每次與醫療保健提供者會面時都會測量患者的頭圍，並根據軟骨發育不全特定的監測標準進行繪製。
椎間孔狹窄：美國小兒科會建議 FMS 患者先進行睡眠研究（多導睡眠圖），如果呈陽性，則進行電腦斷層掃描或 MRI 檢查頸椎區域的壓迫性脊髓型頸椎病變。
胸腰椎後凸 (TLK)：TLK 通常發生在新生兒中，90% 的受影響兒童在 18 個月大時會自行恢復，因為他們的行走能力隨著身體軀幹肌肉的發展而提高。

骨骼發育不良臨床試驗

2023 年 5 月，Nemours Children's Health 將作為美國國立衛生研究院 (FNIH) 加速藥物合作夥伴關係®客製化基因治療聯盟基金會的成員，對 Morquio A 症候群進行突破性的基因治療臨床試驗。
2023 年 3 月，BridgeBio Pharma, Inc. 報告了 PROPEL2 的積極結果，這是一項針對軟骨發育不全兒童的臨床實驗藥物 infigratinib 的 2 期研究。 BridgeBio Pharma, Inc 是一家商業階段的生物製藥公司，專注於遺傳疾病和癌症領域。這些結果顯示該藥物具有一流的療效和較低的副作用風險。

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

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