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한글목차
산필리포 증후군 시장 규모는 향후 몇 년 동안 빠르게 성장할 것으로 예상되며, 2029년까지 연평균 10.5%의 성장률로 137억 9,000만 달러에 달할 것으로 예상됩니다. 예측 기간 동안 예상되는 성장은 산필리포 증후군의 맞춤 치료 전략의 확대, 잠재적 치료 접근법으로서 유전자 치료의 채택 확대, 표적 약물전달 시스템에 대한 수요 증가, 효능이 개선된 줄기세포 치료의 채택 확대, 신생아 스크리닝 프로그램의 전 세계적 확대 등 여러 가지 요인에 기인하고 있습니다. 예측 기간 동안 주요 동향으로는 유전자 치료 기술의 발전, 줄기세포 치료의 혁신, 표적 약물전달 시스템 개발, 혈액뇌장벽(BBB) 투과 전략의 개선, 신경 보호 소분자의 발전 등이 있습니다.
유전자 치료에 대한 관심이 높아짐에 따라 향후 몇 년 동안 산필리포 증후군 시장의 성장을 견인할 것으로 예상됩니다. 유전자 치료는 질병을 치료하거나 예방하기 위해 비정상적인 유전자를 교정하거나 대체하는 것을 목표로 하는 치료를 포함합니다. 이러한 치료법에 대한 관심이 높아지는 이유는 분자 수준에서 근본적인 원인을 타겟팅함으로써 유전성 질환, 희귀질환, 만성질환을 치료할 수 있는 가능성이 있기 때문입니다. 구체적으로, 유전자 치료는 결함이 있는 유전자의 기능적 버전을 제공하고, 효소 생산을 회복시키며, 세포 내 유해 물질의 축적을 감소시킴으로써 산필리포 증후군 치료에 도움이 될 수 있습니다. 예를 들어, 2024년 1월 미국 유전자 및 세포 치료 단체인 미국 유전자 및 세포 치료학회(American Society of Gene &Cell Therapy)는 4분기에 임상 3상 시험 중인 유전자 치료제의 수가 10% 증가했다고 보고했습니다. 이러한 유전자 치료에 대한 관심이 높아지면서 산필리포 증후군 시장의 성장을 견인하고 있습니다.
산필리포 증후군 시장의 주요 기업들은 환자의 예후를 개선하고 질병의 진행을 늦추기 위해 유전자 치료와 같은 획기적인 치료법 개발에 주력하고 있습니다. 유전자 치료는 체내의 결함 유전자를 수정하거나 대체함으로써 유전자를 사용하여 질병을 치료, 예방, 치유하는 혁신적인 의료 접근법입니다. 예를 들어, 2025년 2월 미국의 바이오 제약회사 Ultragenyx Pharmaceutical Inc.는 A형(MPS IIIA)에 대한 AAV 유전자 치료제 UX111(ABO-102)에 대한 새로운 연구 결과를 발표하였습니다. 이 치료는 인지, 수용적 의사소통, 표현적 의사소통의 Bayley-III raw score에서 통계적으로 유의미한 개선을 보였습니다. 또한, 뇌척수액(CSF)의 헤파란황산(HS) 농도가 실질적이고 지속적으로 감소하여 임상적 결과를 개선하는 것으로 나타났습니다.
목차
제1장 주요 요약
제2장 시장 특징
제3장 시장 동향과 전략
제4장 시장 - 금리, 인플레이션, 지정학, 무역 전쟁과 관세, 그리고 코로나와 회복이 시장에 미치는 영향을 포함한 거시경제 시나리오
제5장 세계의 성장 분석과 전략 분석 프레임워크
세계 산필리포 증후군 PESTEL 분석(정치, 사회, 기술, 환경, 법적 요인, 촉진요인과 억제요인)
최종 이용 산업 분석
세계의 산필리포 증후군 시장 : 성장률 분석
세계의 산필리포 증후군 시장 실적 : 규모와 성장, 2019-2024
세계의 산필리포 증후군 시장 예측 : 규모와 성장, 2024-2029, 2034F
세계 산필리포 증후군 전체 시장(TAM)
제6장 시장 세분화
세계의 산필리포 증후군 시장 치료별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
효소보충요법
유전자 치료
게니스테인
기타 치료법
세계의 산필리포 증후군 시장 진단별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
글리코사미노글리칸 분석(GAG 분석)
유전체 데옥시리보핵산 배열 해석(유전체 DNA 배열 해석)
활성 어세이
기타 진단
세계의 산필리포 증후군 시장 : 최종사용자별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
병원
조사기관
세계의 산필리포 증후군 시장 효소보충요법 종류별 하위 세분화, 실적과 예측, 2019-2024, 2024-2029F, 2034F
정맥내 효소보충요법
척수내 효소보충요법
뇌실내(ICV) 효소보충요법
세계의 산필리포 증후군 시장 유전자 치료 종류별 하위 세분화, 실적과 예측, 2019-2024, 2024-2029F, 2034F
아데노 부속 바이러스(AAV)를 이용한 생체내 유전자 치료
렌티바이러스 벡터를 이용한 체외 유전자 치료
비바이러스 벡터 유전자 전달
세계의 산필리포 증후군 시장 게니스테인 종류별 하위 세분화, 실적과 예측, 2019-2024, 2024-2029F, 2034F
경구 게니스테인 보급
고용량 게니스테인 요법
저용량 게니스테인 요법
세계의 산필리포 증후군 시장, 기타 치료법 하위 세분화, 유형별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
기질 환원 요법(SRT)
줄기세포 치료
항염증제
대증요법약
제7장 지역별·국가별 분석
세계의 산필리포 증후군 시장 : 지역별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
세계의 산필리포 증후군 시장 : 국가별, 실적과 예측, 2019-2024, 2024-2029F, 2034F
제8장 아시아태평양 시장
제9장 중국 시장
제10장 인도 시장
제11장 일본 시장
제12장 호주 시장
제13장 인도네시아 시장
제14장 한국 시장
제15장 서유럽 시장
제16장 영국 시장
제17장 독일 시장
제18장 프랑스 시장
제19장 이탈리아 시장
제20장 스페인 시장
제21장 동유럽 시장
제22장 러시아 시장
제23장 북미 시장
제24장 미국 시장
제25장 캐나다 시장
제26장 남미 시장
제27장 브라질 시장
제28장 중동 시장
제29장 아프리카 시장
제30장 경쟁 구도와 기업 개요
산필리포 증후군 시장 : 경쟁 구도
산필리포 증후군 시장 : 기업 개요
Takeda Pharmaceutical Company Limited Overview, Products and Services, Strategy and Financial Analysis
BioMarin Pharmaceutical Inc. Overview, Products and Services, Strategy and Financial Analysis
Qiagen N.V. Overview, Products and Services, Strategy and Financial Analysis
Natera Inc. Overview, Products and Services, Strategy and Financial Analysis
Guardant Health Inc. Overview, Products and Services, Strategy and Financial Analysis
제31장 기타 주요 기업과 혁신적 기업
Novogene Corporation
Ultragenyx Pharmaceutical Inc.
JCR Pharmaceuticals Co. Ltd.
Oxford Nanopore Technologies plc
Pacific Biosciences of California Inc.
Denali Therapeutics Inc.
Macrogen Inc.
REGENXBIO Inc.
Personalis Inc.
Veritas Genetics Inc.
Greenwood Genetic Center
ArmaGen Inc.
Abeona Therapeutics Inc.
Orchard Therapeutics plc
Allievex Corporation
제32장 세계의 시장 경쟁 벤치마킹과 대시보드
제33장 주요 인수합병
제34장 최근의 시장 동향
제35장 시장 잠재력이 높은 국가, 부문, 전략
산필리포 증후군 시장 2029 : 새로운 기회를 제공하는 국가
산필리포 증후군 시장 2029 : 새로운 기회를 제공하는 부문
산필리포 증후군 시장 2029 : 성장 전략
시장 동향에 기반한 전략
경쟁 전략
제36장 부록
ksm
영문 목차
영문목차
Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is a rare genetic disorder caused by enzyme deficiencies that hinder the body's ability to break down heparan sulfate, a complex sugar. This leads to the accumulation of toxic substances in cells, particularly affecting the central nervous system. The disease is characterized by progressive neurodegeneration, developmental delays, behavioral problems, and a decline in motor and cognitive functions.
Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.
The primary treatments for Sanfilippo syndrome include enzyme replacement therapy (ERT), gene therapy, genistein, and other therapeutic options. ERT supplements the deficient or faulty enzymes to reduce the buildup of harmful substances in individuals with the condition. Diagnostic methods for Sanfilippo syndrome include glycosaminoglycan (GAG) analysis, genomic DNA sequencing, activity assays, and other techniques. These diagnostics are primarily utilized by various end users, such as hospitals and research institutions.
The sanfilippo syndrome market research report is one of a series of new reports from The Business Research Company that provides sanfilippo syndrome market statistics, including the sanfilippo syndrome industry global market size, regional shares, competitors with the sanfilippo syndrome market share, detailed sanfilippo syndrome market segments, market trends, and opportunities, and any further data you may need to thrive in the sanfilippo syndrome industry. This sanfilippo syndrome market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The sanfilippo syndrome market size has grown rapidly in recent years. It will grow from$8.33 billion in 2024 to $9.24 billion in 2025 at a compound annual growth rate (CAGR) of 10.8%. The growth observed in the historic period can be attributed to several factors, including increased awareness of rare genetic disorders, rising government funding for rare disease research, the expansion of patient advocacy groups and support organizations, the growth of genetic screening programs, and the increase in orphan drug designations for rare diseases.
The sanfilippo syndrome market size is expected to see rapid growth in the next few years. It will grow to$13.79 billion in 2029 at a compound annual growth rate (CAGR) of 10.5%. The growth projected for the forecast period can be attributed to several factors, including the expansion of personalized medicine strategies for Sanfilippo syndrome, increasing adoption of gene therapy as a potential curative approach, rising demand for targeted drug delivery systems, greater adoption of stem cell therapies with improved efficacy, and the global expansion of newborn screening programs. Key trends in the forecast period include advancements in gene therapy technologies, innovations in stem cell therapy, development of targeted drug delivery systems, improvements in blood-brain barrier (BBB) penetration strategies, and progress in neuroprotective small molecules.
The growing emphasis on gene therapies is expected to drive the expansion of the Sanfilippo syndrome market in the coming years. Gene therapies involve medical treatments that aim to modify or replace faulty genes to treat or prevent diseases. The increasing focus on these therapies is due to their potential to address genetic disorders, rare diseases, and chronic conditions by targeting the root causes at the molecular level. Specifically, gene therapies are beneficial in treating Sanfilippo syndrome by delivering functional versions of the defective gene to restore enzyme production, thus reducing harmful substance buildup in cells. For example, in January 2024, the American Society of Gene & Cell Therapy, a U.S.-based organization for gene and cell therapy, reported a 10% increase in the number of gene therapies in Phase III clinical trials during the fourth quarter, marking the first quarterly growth of this kind since the third quarter of 2022. This growing emphasis on gene therapies is, therefore, fueling the growth of the Sanfilippo syndrome market.
Leading companies in the Sanfilippo syndrome market are focusing on developing groundbreaking treatments, such as gene therapies, to enhance patient outcomes and slow the progression of the disease. Gene therapy is an innovative medical approach that uses genes to treat, prevent, or cure diseases by correcting or replacing defective genes within the body. For instance, in February 2025, Ultragenyx Pharmaceutical Inc., a U.S.-based biopharmaceutical company, announced new findings related to UX111 (ABO-102), an AAV gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The treatment showed statistically significant improvements in Bayley-III raw scores for cognition, receptive communication, and expressive communication. Additionally, it resulted in a substantial and sustained reduction in cerebrospinal fluid (CSF) heparan sulfate (HS) levels, which was linked to improved clinical outcomes.
In May 2022, Ultragenyx Pharmaceutical Inc. acquired the global rights to the AAV-based gene therapy ABO-102 from Abeona Therapeutics Inc. for an undisclosed amount. This acquisition granted Ultragenyx full control over ABO-102 (now referred to as UX111), a gene therapy for Sanfilippo syndrome Type A (MPS IIIA). Under the terms of the agreement, Abeona remains eligible for tiered royalties of up to 10% on net sales, as well as commercial milestone payments upon regulatory approval. Abeona Therapeutics Inc. is a U.S.-based clinical-stage biopharmaceutical company specializing in AAV-based gene therapies for neuromuscular and lysosomal storage disorders.
Major players in the sanfilippo syndrome market are Takeda Pharmaceutical Company Limited, BioMarin Pharmaceutical Inc., Qiagen N.V., Natera Inc., Guardant Health Inc., Novogene Corporation, Ultragenyx Pharmaceutical Inc., JCR Pharmaceuticals Co. Ltd., Oxford Nanopore Technologies plc, Pacific Biosciences of California Inc., Denali Therapeutics Inc., Macrogen Inc., REGENXBIO Inc., Personalis Inc., Veritas Genetics Inc., Greenwood Genetic Center, ArmaGen Inc., Abeona Therapeutics Inc., Orchard Therapeutics plc, and Allievex Corporation.
North America was the largest region in the sanfilippo syndrome market in 2024. The regions covered in sanfilippo syndrome report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the sanfilippo syndrome market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The sanfilippo syndrome market consists of revenues earned by entities by providing services such as substrate reduction therapies (SRT), and other emerging treatment modalities and related diagnostic services, supportive care solutions, and clinical trial services. The market value includes the value of related goods sold by the service provider or included within the service offering. The sanfilippo syndrome market also includes sales of antiepileptic drugs, sleep aids, and behavioral management medications. Values in this market are 'factory gate' values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
Sanfilippo Syndrome Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses on sanfilippo syndrome market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Where is the largest and fastest growing market for sanfilippo syndrome ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The sanfilippo syndrome market global report from the Business Research Company answers all these questions and many more.
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.
The market characteristics section of the report defines and explains the market.
The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
The forecasts are made after considering the major factors currently impacting the market. These include:
The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
Market segmentations break down the market into sub markets.
The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:1) By Treatment: Enzyme Replacement Therapy; Gene Therapy; Genistein; Other Treatments
2) By Diagnosis: Glycosaminoglycan Analysis (GAG Analysis); Genomic Deoxyribonucleic Acid Sequencing (Genomic DNA Sequencing); Activity Assay; Other Diagnosis
2) By Gene Therapy: Adeno-Associated Virus (AAV)-Based In Vivo Gene Therapy; Lentiviral Vector-Based Ex Vivo Gene Therapy; Non-Viral Vector Gene Delivery
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita,
Data segmentations: country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Table of Contents
1. Executive Summary
2. Sanfilippo Syndrome Market Characteristics
3. Sanfilippo Syndrome Market Trends And Strategies
4. Sanfilippo Syndrome Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market
5. Global Sanfilippo Syndrome Growth Analysis And Strategic Analysis Framework
5.1. Global Sanfilippo Syndrome PESTEL Analysis (Political, Social, Technological, Environmental and Legal Factors, Drivers and Restraints)
5.2. Analysis Of End Use Industries
5.3. Global Sanfilippo Syndrome Market Growth Rate Analysis
5.4. Global Sanfilippo Syndrome Historic Market Size and Growth, 2019 - 2024, Value ($ Billion)
5.5. Global Sanfilippo Syndrome Forecast Market Size and Growth, 2024 - 2029, 2034F, Value ($ Billion)
5.6. Global Sanfilippo Syndrome Total Addressable Market (TAM)
6. Sanfilippo Syndrome Market Segmentation
6.1. Global Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
Enzyme Replacement Therapy
Gene Therapy
Genistein
Other Treatments
6.2. Global Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
Glycosaminoglycan Analysis (GAG Analysis)
Genomic Deoxyribonucleic Acid Sequencing (Genomic DNA Sequencing)
Activity Assay
Other Diagnosis
6.3. Global Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
Hospitals
Research Institutions
6.4. Global Sanfilippo Syndrome Market, Sub-Segmentation Of Enzyme Replacement Therapy, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
