세계의 골격이형성증 시장 : 예측(2024-2029년)

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