表紙:クローブス症候群の世界市場-2023年~2030年
市場調査レポート
商品コード
1372607

クローブス症候群の世界市場-2023年~2030年

Global CLOVES Syndrome Market - 2023-2030

出版日: | 発行: DataM Intelligence | ページ情報: 英文 160 Pages | 納期: 約2営業日

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クローブス症候群の世界市場-2023年~2030年
出版日: 2023年10月18日
発行: DataM Intelligence
ページ情報: 英文 160 Pages
納期: 約2営業日
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
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  • 概要
  • 目次
概要

概要

先天性脂肪腫性過成長、血管奇形、表皮母斑、脊椎/骨格異常/膠原病(クローブス)症候群は、血管(血管が関与する)、皮膚、脊椎、骨または関節の異常の組み合わせを特徴とする非常にまれな先天性(出生時に存在する)疾患です。

クローブス症候群は、現在までに世界中で200例未満しか確認されていないです。クローブス症候群はPIK3CAと呼ばれる遺伝子の突然変異によって引き起こされます。クローブス症候群の子どもの長期的な見通しは、年齢と症状の重症度によって異なります。

クローブス症候群はまれであり、症状も様々であるため、診断が難しい場合があります。臨床的評価、遺伝子検査、医療用画像診断、様々な特徴の注意深い評価を組み合わせて、決定的な診断を下すことがよくあります。クローブス症候群の管理には集学的アプローチが必要で、身体的異常に対処するための手術、血管奇形を管理するための介入、関連合併症に対処するための支持療法などが含まれます。病状の進行をモニターし、新たな問題に対処するためには、定期的な経過観察が不可欠です。

さらに、クローブス症候群の調査における資金提供の増加、希少な遺伝性疾患に対する医療従事者や一般市民の意識の高まりが、予測期間中に市場を牽引すると予想される要因です。

力学

クローブス症候群のゼブラフィッシュモデルへの資金提供の増加が市場成長を促進すると予想される

脂肪細胞の過剰増殖、血管奇形、その他の身体的異常を引き起こす遺伝性疾患であるクローブスのゼブラフィッシュモデル開発への投資は、この稀な疾患の研究と潜在的治療における大きな進歩です。

2022年10月20日、エディンバラ大学のパットン研究室は、クローブス症候群のゼブラフィッシュモデルを作成するために10万米ドルの研究寄付を受けた。このプロジェクトの主な目的は、ゼブラフィッシュの助けを借りて、クローブス症候群の新鮮で臨床的に重要な動物モデルを確立することです。

ゼブラフィッシュはヒトの病気を研究するモデルとして広く受け入れられています。ヒトの病気を引き起こす遺伝子の少なくとも70%は、ゼブラフィッシュに対応するものがあります。この調査をさらに進めるため、科学者たちはCRISPR-Cas9技術を用いて、クローブス症候群の原因となる1次変異を、正常な遺伝子とともにゼブラフィッシュのPik3caに導入する予定です。蛍光マーカーを用いて、Pik3ca変異細胞の働きや周囲の正常細胞との相互作用を観察する予定です。

さらに研究者たちは、この方法によって、これまで使用されてきた他の動物モデルと比較して、より詳細なクローブス症候群のモデルを作成できると確信しています。このモデルは、クローブス症候群の新しい治療法をスクリーニングするための基礎として使用することができます。Liz Patton博士はこのプロジェクトを率いており、メラノーマ細胞集団を同定し標的とするための主要なツールとしてゼブラフィッシュモデルを利用しています。

このように、上記の要因から、予測期間中、市場は牽引役となることが期待されます。

一般市民におけるクローブ症候群の認知度向上が市場成長を促進する見込み

クローブス症候群啓発デーは、身体の様々な部位の発達に影響を及ぼす稀な遺伝性疾患についての認識を広めるために毎年8月3日に制定されています。この日は、クローブス症候群に罹患した個人や家族に対する理解を促進し、支援と連帯を広げることを目的としています。

コミュニティは団結し、クローブス症候群に関する情報やリソースを交換し、初期症状の発見について個人を教育します。ワークショップでは、社会的包摂と、罹患者を持つ家族を支援する社会の責任についても取り上げます。この病気は、記録されている症例がわずか200例強しかない希少な病気であるため、この病気に関する資金援助や調査が十分に行われていないです。その結果、罹患した家族は有効な治療法にアクセスできず、認知度が向上しなければ必要な支援を受けられないかもしれないです。

クローブス症候群啓発デーは、クローブス症候群とその生活への影響に関する情報とリソースを広める機会を提供します。この日は、地域社会がこの病気と闘うために知識や資源を出し合う日でもあります。以上のような要因から、予測期間中、市場は拡大すると予想されます。

クローブス症候群の管理に伴う課題が市場成長の妨げになる見込み

National Organization for Rare Disorders, Inc.によると、クローブス症候群の管理は非常に困難であり、過成長障害や血管異常の経験を持つ医師による学際的なチームが必要です。

例えば、クローブス症候群の症状は様々で、脂肪組織の塊、足や手の問題、拡大した静脈、アザ、脊椎湾曲、繋索などがあります。これらの症状を管理し、血栓などの合併症を予防するには、継続的な医学的・外科的介入が必要です。したがって、クローブス症候群の管理に関連する課題により、予測期間中の市場成長の妨げになると予想されます。

このレポートの詳細情報-サンプル請求

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

  • 影響要因
    • 促進要因
      • クローブス症候群のゼブラフィッシュモデルへの資金提供の増加
      • 一般市民のクローブス症候群に対する認識の高まり
    • 抑制要因
      • クローブス症候群の管理に伴う課題
    • 機会
      • ゲノミクスと遺伝子配列決定技術の進歩
    • 影響分析

第5章 産業分析

  • ポーターの5フォース分析
  • サプライチェーン分析
  • 価格分析
  • 規制分析
  • パイプライン分析
  • アンメットニーズ
  • DMI意見

第6章 COVID-19分析

第7章 治療タイプ別

  • 硬化療法
  • 塞栓術
  • デバルキング手術
  • 薬物療法
  • リハビリ治療
  • その他

第8章 販売チャネル別

  • エンドユーザー
    • 病院
    • 外来手術センター
    • 作業療法センター
    • その他
  • 流通チャネル
    • 病院薬局
    • 小売薬局
    • オンライン薬局

第9章 地域別

  • 北米
    • 米国
    • カナダ
    • メキシコ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他の欧州
  • 南米
    • ブラジル
    • アルゼンチン
    • その他の南米
  • アジア太平洋
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他のアジア太平洋
  • 中東およびアフリカ

第10章 競合情勢

  • 競合シナリオ
  • 製品ベンチマーク
  • 企業シェア分析
  • 主な発展と戦略

第11章 企業プロファイル

  • Pfizer Inc.
  • Novartis AG
  • Glenmark Pharmaceuticals U.S. Inc., USA
  • Methapharm, Inc.
  • Apotex Inc.
  • AngioDynamics
  • ENDO-FLEX GmbH
  • C.R. Bard, Inc
  • Cook Medical LLC
  • Cordis

第12章 付録

目次
Product Code: PH7306

Overview

Congenital Lipomatous Overgrowth, Vascular Malformations, Epidermal Nevis, Spinal/Skeletal Anomalies/Scoliosis (CLOVES) syndrome is a very rare congenital (present at birth) disorder characterized by a combination of vascular (involving the blood vessels), skin, spinal, and bone or joint abnormalities.

Fewer than 200 cases of CLOVES syndrome have been identified worldwide to date. CLOVES are caused by mutations in a gene called PIK3CA. The long-term outlook for a child with CLOVES will depend on their age and the severity of their symptoms.

Diagnosing CLOVES syndrome can be difficult as it is rare and symptoms can vary. Clinical assessment, genetic testing, medical imaging, and careful assessment of the various features are frequently combined to make a conclusive diagnosis. Managing CLOVES syndrome requires a multidisciplinary approach, which may involve surgery to address physical abnormalities, interventions to manage vascular malformations, and supportive therapies to address associated complications. Regular medical follow-ups are essential to monitor the progression of the condition and address any emerging issues.

Furthermore, the increasing funding in the research of CLOVES Syndrome, and increasing awareness among medical professionals and the general public about rare genetic disorders are the factors expected to drive the market over the forecast period.

Dynamics

Increasing Funding in Zebrafish Models of CLOVES Syndrome is Expected to Drive the Market Growth

Investing in the development of zebrafish models for CLOVES, a genetic disorder that causes overgrowth of fat cells, vascular malformations, and other physical anomalies, is a major advancement in the research and potential treatment of this rare condition.

On October 20, 2022, the Patton Lab at the University of Edinburgh was granted a research donation of $100,000 to create zebrafish models of CLOVES syndrome. The main objective of this project is to establish a fresh and clinically significant animal model of CLOVES syndrome with the help of zebrafish.

Zebrafish have been widely accepted as models for studying human diseases. At least 70% of genes that cause human diseases have counterparts in zebrafish. To further this research, scientists intend to employ CRISPR-Cas9 technology to introduce the primary mutation responsible for CLOVES syndrome, along with the normal gene, into zebrafish Pik3ca. Using fluorescent markers, the scientists will observe the actions of Pik3ca-mutant cells and how they interact with the surrounding normal cells.

In addition, researchers are confident that this method will enable them to create a more detailed model of CLOVES syndrome compared to other animal models used previously. This model can be used as a foundation for screening new treatments for CLOVES syndrome. Dr. Liz Patton leads this project and utilizes zebrafish models as her primary tool for identifying and targeting melanoma cell populations.

Thus, owing to the above factors, the market is expected to drive over the forecast period.

Increasing Awareness of CLOVE Syndrome among the General Public is Expected to Drive the Market Growth

Cloves Syndrome Awareness Day is observed on August 3 every year to spread awareness about the rare genetic disorder that affects the development of many different parts of the body. The day aims to foster understanding and extend support, and solidarity for individuals and families affected by CLOVES syndrome.

Communities unite to exchange information and resources regarding CLOVES disease and educate individuals on identifying early symptoms. Workshops also address social inclusion and society's responsibility in assisting families with afflicted members. As it is a rare disease with only slightly over 200 documented cases, there is insufficient funding and research allocated towards it. As a result, affected families lack access to effective treatments and may not receive the support they require without increased awareness.

Therefore, Cloves Syndrome Awareness Day provides an opportunity to spread information and resources about CLOVES syndrome and its impact on people's lives. It is a day for communities to pool their knowledge and resources to combat the disease. Thus, owing to the above factors, the market is expected to drive over the forecast period.

Challenges Associated with Managing CLOVES Syndrome is Expected to Hamper the Market Growth

According to National Organization for Rare Disorders, Inc., managing CLOVES syndrome can be very challenging and requires an interdisciplinary team of physicians with experience in overgrowth disorders and vascular anomalies.

For instance, the symptoms of CLOVES syndrome can vary widely and may include fatty tissue masses, problems with feet and hands, dilated veins, birthmarks, spinal curvature, and tethered cord. Managing these symptoms and preventing complications such as blood clots requires ongoing medical and surgical interventions. Thus, owing to the challenges associated with the management of CLOVE syndrome is expected to hamper the market growth over the forecast period.

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Segment Analysis

The global CLOVES syndrome market is segmented based on treatment type, sales channel and region.

Owing to the Increasing FDA Approvals and Rising Acceleration of Treatment Options, the Treatment Type Segment Accounted for Approximately 39.4% of the CLOVE Syndrome Market Share

CLOVES syndrome is a condition that is present from birth, and it is characterized by excessive growth of tissue and the presence of complicated vascular irregularities. When the FDA approves treatment for this condition, it represents a significant milestone in the development of therapies for CLOVES Syndrome. This approval not only confirms that the treatment is safe and effective, but it also has far-reaching effects on patient access, market growth, investment, and research initiatives.

For instance, on April 6, 2022, Novartis announced the U.S. Food and Drug Administration (FDA) granted accelerated approval to Vijoice (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy.

Vijoice is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million. Following the Accelerated Approval Program, continued approval may be contingent upon verification and description of clinical benefit from confirmatory evidence.

Hence, owing to the above factors, the market segment is expected to hold the largest market share over the forecast period.

Geographical Penetration

North America Accounted for Approximately 39.2% of the Market Share in 2022, Owing to the Increasing Funding, and Availability of Targeted Treatments

North America is expected to hold the largest market share owing to the increasing funding, availability of targeted treatments, and increasing prevalence of spinal problems.

For instance, in May 2022, Dr. Wen Yih Aw of The University of North Carolina/Chapel Hill has been awarded a $50,000 grant by the Scientific and Medical Advisory Board and Board of Directors of the CLOVES Syndrome Community for her project titled Non-canonical Signaling and therapeutic targets for vascular anomalies in CLOVES.

The objective of this project is to gain a better understanding of the formation of vascular malformations and identify the genes and proteins that could potentially be targeted to reverse them and promote healthy vasculature. Dr. Aw has created a micro-physiological model using human cells to investigate vascular malformations. Her research has demonstrated that this model can replicate the development of vascular malformations when using cells expressing a mutated version of the PIK3CA gene, which is associated with CLOVES.

Dr. Aw's project involves testing two drugs that have been effective in treating other types of vascular malformations and certain cancers. Each drug targets a specific protein, and by testing them in her micro-physiological model, she can observe how inhibiting each protein affects individual cells and blood vessel formation.

The results of her work not only have the potential to explore the use of these drugs as treatments for CLOVES-related vascular malformations but also to enhance our understanding of how these malformations develop. This could ultimately lead to the development of new drugs and interventions. Thus, owing to the above factors, the North American region is expected to hold the largest market share over the forecast period.

Competitive Landscape

The major global players in the market include: Pfizer Inc., Novartis AG, Glenmark Pharmaceuticals U.S. Inc., USA, Methapharm, Inc., Apotex Inc., AngioDynamics, ENDO-FLEX GmbH, C.R. Bard, Inc, Cook Medical LLC, and Cordis among others.

Key Developments

  • On April 6, 2022, Novartis announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval to Vijoice (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. Vijoice is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million.

COVID-19 Impact Analysis

The COVID-19 pandemic has significantly affected the healthcare industry, including the market for CLOVES Syndrome. Many regions experienced disruptions in healthcare services due to lockdowns, restrictions, and healthcare system strain, which may have caused delays in diagnosis and care for individuals with CLOVES Syndrome, especially those seeking specialized medical attention.

Clinical trials for rare diseases, including CLOVES Syndrome, were also affected by the pandemic. Trials may have been postponed, suspended, or faced challenges in recruiting participants and conducting in-person assessments. The COVID-19-related research redirection could have impacted the funding availability for rare disease studies, potentially affecting the ongoing and planned research efforts for CLOVES Syndrome.

In addition, advocacy organizations and patient support groups may have faced challenges in organizing events, fundraising, and raising awareness due to restrictions on gatherings and reduced attention to non-COVID-19 health issues.

Furthermore, remote work became more prevalent, which could have caused challenges for researchers and clinicians in conducting collaborative research, accessing labs, and performing hands-on experiments for understanding and treating CLOVES Syndrome. Thus, owing to the above factors, the global market for CLOVES Syndrome is anticipated to have a negative impact on the market.

By Treatment Type

  • Sclerotherapy
  • Embolization
  • Debulking Surgery
  • Medications
  • Rehabilitation Therapies
  • Others

By Sales Channel

  • End User
    • Hospitals
    • Ambulatory Surgical Center
    • Occupational Therapy Centers
    • Others
  • Distribution Channel
    • Hospital Pharmacies
    • Retail Pharmacies
    • Online Pharmacies

By Region

  • North America
    • U.S.
    • Canada
    • Mexico
  • Europe
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Rest of Europe
  • South America
    • Brazil
    • Argentina
    • Rest of South America
  • Asia-Pacific
    • China
    • India
    • Japan
    • Australia
    • Rest of Asia-Pacific
  • Middle East and Africa

Why Purchase the Report?

  • To visualize the global CLOVES syndrome market segmentation based on treatment type, sales channel and region as well as understand key commercial assets and players
  • Identify commercial opportunities by analyzing trends and co-development
  • Excel data sheet with numerous data points of global CLOVES syndrome market-level with all segments
  • PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study
  • Product mapping available as Excel consisting of key products of all the major players

The global CLOVES syndrome market report would provide approximately 53 tables, 49 figures, and 160 Pages.

Target Audience 2023

  • Manufacturers/ Buyers
  • Industry Investors/Investment Bankers
  • Research Professionals
  • Emerging Companies

Table of Contents

1. Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

  • 3.1. Snippet by Treatment Type
  • 3.2. Snippet by Sales Channel
  • 3.3. Snippet by Region

4. Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Increasing Funding in Zebrafish Models of CLOVES Syndrome
      • 4.1.1.2. Increasing Awareness of CLOVE Syndrome among the General Public
    • 4.1.2. Restraints
      • 4.1.2.1. Challenges Associated with Managing CLOVES Syndrome
    • 4.1.3. Opportunity
      • 4.1.3.1. Advancements in Genomics and Genetic Sequencing Technologies
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's 5 Forces Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Pricing Analysis
  • 5.4. Regulatory Analysis
  • 5.5. Pipeline Analysis
  • 5.6. Unmet Needs
  • 5.7. DMI Opinion

6. COVID-19 Analysis

  • 6.1. Analysis of COVID-19
    • 6.1.1. Scenario Before COVID-19
    • 6.1.2. Scenario During COVID-19
    • 6.1.3. Scenario Post COVID-19
  • 6.2. Pricing Dynamics Amid COVID-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During the Pandemic
  • 6.5. Manufacturers' Strategic Initiatives
  • 6.6. Conclusion

7. By Treatment Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 7.1.2. Market Attractiveness Index, By Treatment Type
  • 7.2. Sclerotherapy*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%
  • 7.3. Embolization
  • 7.4. Debulking Surgery
  • 7.5. Medication
  • 7.6. Rehabilitation Therapies
  • 7.7. Others

8. By Sales Channel

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Sales Channel
    • 8.1.2. Market Attractiveness Index, By Sales Channel
  • 8.2. End User*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
      • 8.2.2.1. Hospitals
      • 8.2.2.2. Ambulatory Surgical Centers
      • 8.2.2.3. Occupational Therapy Centers
      • 8.2.2.4. Others
  • 8.3. Distribution Channel
      • 8.3.1.1. Hospital Pharmacies
      • 8.3.1.2. Retail Pharmacies
      • 8.3.1.3. Online Pharmacies

9. By Region

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
    • 9.1.2. Market Attractiveness Index, By Region
  • 9.2. North America
    • 9.2.1. Introduction
    • 9.2.2. Key Region-Specific Dynamics
    • 9.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Sales Channel
    • 9.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.2.5.1. U.S.
      • 9.2.5.2. Canada
      • 9.2.5.3. Mexico
  • 9.3. Europe
    • 9.3.1. Introduction
    • 9.3.2. Key Region-Specific Dynamics
    • 9.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Sales Channel
    • 9.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.3.5.1. Germany
      • 9.3.5.2. U.K.
      • 9.3.5.3. France
      • 9.3.5.4. Italy
      • 9.3.5.5. Spain
      • 9.3.5.6. Rest of Europe
  • 9.4. South America
    • 9.4.1. Introduction
    • 9.4.2. Key Region-Specific Dynamics
    • 9.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Sales Channel Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.4.4.1. Brazil
      • 9.4.4.2. Argentina
      • 9.4.4.3. Rest of South America
  • 9.5. Asia Pacific
    • 9.5.1. Introduction
    • 9.5.2. Key Region-Specific Dynamics
    • 9.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Sales Channel
    • 9.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.5.5.1. China
      • 9.5.5.2. India
      • 9.5.5.3. Japan
      • 9.5.5.4. Australia
      • 9.5.5.5. Rest of Asia Pacific
  • 9.6. Middle East and Africa
    • 9.6.1. Introduction
    • 9.6.2. Key Region-Specific Dynamics
    • 9.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Sales Channel

10. Competitive Landscape

  • 10.1. Competitive Scenario
  • 10.2. Product Benchmarking
  • 10.3. Company Share Analysis
  • 10.4. Key Developments and Strategies

11. Company Profiles

  • 11.1. Pfizer Inc.*
    • 11.1.1. Company Overview
    • 11.1.2. Product Portfolio and Description
    • 11.1.3. Financial Overview
    • 11.1.4. Key Developments
  • 11.2. Novartis AG
  • 11.3. Glenmark Pharmaceuticals U.S. Inc., USA
  • 11.4. Methapharm, Inc.
  • 11.5. Apotex Inc.
  • 11.6. AngioDynamics
  • 11.7. ENDO-FLEX GmbH
  • 11.8. C.R. Bard, Inc
  • 11.9. Cook Medical LLC
  • 11.10. Cordis

LIST NOT EXHAUSTIVE

12. Appendix

  • 12.1. About Us and Services
  • 12.2. Contact Us