サンフィリッポ症候群の世界市場レポート 2025年

サンフィリッポ症候群市場規模は、今後数年で急成長が見込まれます。2029年には137億9,000万米ドルに成長し、CAGRは10.5%となります。予測期間に予測される成長は、サンフィリッポ症候群の個別化医療戦略の拡大、潜在的な治療アプローチとしての遺伝子治療の採用拡大、標的ドラッグデリバリーシステムに対する需要の高まり、有効性が向上した幹細胞治療の採用拡大、新生児スクリーニングプログラムの世界的拡大など、いくつかの要因に起因しています。予測期間における主な動向としては、遺伝子治療技術の発展、幹細胞治療の革新、標的ドラッグデリバリーシステムの開発、血液脳関門（BBB）浸透戦略の改善、神経保護小分子の進歩などが挙げられます。

遺伝子治療への注目の高まりは、今後数年間のサンフィリッポ症候群市場の拡大を牽引すると予想されます。遺伝子治療には、病気の治療や予防のために異常な遺伝子を修正したり置き換えたりすることを目的とした治療が含まれます。これらの治療法への注目が高まっているのは、分子レベルでの根本的な原因をターゲットにすることで、遺伝性疾患、希少疾患、慢性疾患に対処できる可能性があるためです。具体的には、遺伝子治療は、欠陥のある遺伝子の機能的バージョンを提供し、酵素産生を回復させ、細胞内の有害物質の蓄積を減少させることにより、サンフィリッポ症候群の治療に有益です。例えば、2024年1月、米国を拠点とする遺伝子・細胞治療団体である米国遺伝子・細胞治療学会（American Society of Gene &Cell Therapy）は、第4四半期に第III相臨床試験中の遺伝子治療薬の数が10％増加したと報告しました。このような遺伝子治療への注目の高まりが、サンフィリッポ症候群市場の成長を後押ししています。

サンフィリッポ症候群市場の主要企業は、患者の予後を改善し、病気の進行を遅らせるために、遺伝子治療などの画期的な治療法の開発に注力しています。遺伝子治療は、体内の欠陥遺伝子を修正または置換することにより、遺伝子を用いて病気を治療、予防、治癒する革新的な医療アプローチです。例えば、2025年2月、米国を拠点とするバイオ医薬品会社であるUltragenyx Pharmaceutical Inc.は、A型（MPS IIIA）に対するAAV遺伝子治療薬UX111（ABO-102）に関する新たな知見を発表しました。この治療により、認知、受容性コミュニケーション、表現性コミュニケーションのBayley-III raw scoreにおいて統計学的に有意な改善が認められました。さらに、脳脊髄液（CSF）のヘパラン硫酸（HS）濃度が実質的かつ持続的に低下し、臨床転帰の改善につながりました。

目次

第1章 エグゼクティブサマリー

第2章 市場の特徴

第3章 市場動向と戦略

第4章 市場- 金利、インフレ、地政学、貿易戦争と関税、そしてコロナ禍と回復が市場に与える影響を含むマクロ経済シナリオ

第5章 世界の成長分析と戦略分析フレームワーク

• 世界サンフィリッポ症候群 PESTEL分析（政治、社会、技術、環境、法的要因、促進要因と抑制要因）
• 最終用途産業の分析
• 世界のサンフィリッポ症候群市場：成長率分析
• 世界のサンフィリッポ症候群市場の実績：規模と成長, 2019-2024
• 世界のサンフィリッポ症候群市場の予測：規模と成長, 2024-2029, 2034F
• 世界サンフィリッポ症候群総アドレス可能市場（TAM）

第6章 市場セグメンテーション

• 世界のサンフィリッポ症候群市場治療別、実績と予測, 2019-2024, 2024-2029F, 2034F
• 酵素補充療法
• 遺伝子治療
• ゲニステイン
• その他の治療法
• 世界のサンフィリッポ症候群市場診断別、実績と予測, 2019-2024, 2024-2029F, 2034F
• グリコサミノグリカン分析（GAG分析）
• ゲノムデオキシリボ核酸配列解析（ゲノムDNA配列解析）
• 活性アッセイ
• その他の診断
• 世界のサンフィリッポ症候群市場：エンドユーザー別、実績と予測, 2019-2024, 2024-2029F, 2034F
• 病院
• 調査機関
• 世界のサンフィリッポ症候群市場酵素補充療法の種類別サブセグメンテーション、実績と予測, 2019-2024, 2024-2029F, 2034F
• 静脈内酵素補充療法
• 脊髄内酵素補充療法
• 脳室内（ICV）酵素補充療法
• 世界のサンフィリッポ症候群市場遺伝子治療の種類別サブセグメンテーション、実績と予測, 2019-2024, 2024-2029F, 2034F
• アデノ随伴ウイルス（AAV）を用いた生体内遺伝子治療
• レンチウイルスベクターを用いた体外遺伝子治療
• 非ウイルスベクター遺伝子送達
• 世界のサンフィリッポ症候群市場ゲニステインの種類別サブセグメンテーション、実績と予測, 2019-2024, 2024-2029F, 2034F
• 経口ゲニステイン補給
• 高用量ゲニステイン療法
• 低用量ゲニステイン療法
• 世界のサンフィリッポ症候群市場、その他の治療法のサブセグメンテーション、タイプ別、実績と予測, 2019-2024, 2024-2029F, 2034F
• 基質還元療法（SRT）
• 幹細胞療法
• 抗炎症剤
• 対症療法薬

第7章 地域別・国別分析

• 世界のサンフィリッポ症候群市場：地域別、実績と予測, 2019-2024, 2024-2029F, 2034F
• 世界のサンフィリッポ症候群市場：国別、実績と予測, 2019-2024, 2024-2029F, 2034F

第8章 アジア太平洋市場

第9章 中国市場

第10章 インド市場

第11章 日本市場

第12章 オーストラリア市場

第13章 インドネシア市場

第14章 韓国市場

第15章 西欧市場

第16章 英国市場

第17章 ドイツ市場

第18章 フランス市場

第19章 イタリア市場

第20章 スペイン市場

第21章 東欧市場

第22章 ロシア市場

第23章 北米市場

第24章 米国市場

第25章 カナダ市場

第26章 南米市場

第27章 ブラジル市場

第28章 中東市場

第29章 アフリカ市場

第30章 競合情勢と企業プロファイル

• サンフィリッポ症候群市場：競合情勢
• サンフィリッポ症候群市場：企業プロファイル
  • 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

第31章 その他の大手企業と革新的企業

• 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

第32章 世界の市場競合ベンチマーキングとダッシュボード

第33章 主要な合併と買収

第34章 最近の市場動向

第35章 市場の潜在力が高い国、セグメント、戦略

• サンフィリッポ症候群市場2029：新たな機会を提供する国
• サンフィリッポ症候群市場2029：新たな機会を提供するセグメント
• サンフィリッポ症候群市場2029：成長戦略
  • 市場動向に基づく戦略
  • 競合の戦略

第36章 付録

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
• 3) By End-User: Hospitals; Research Institutions
• Subsegments:
• 1) By Enzyme Replacement Therapy: Intravenous Enzyme Replacement Therapy; Intrathecal Enzyme Replacement Therapy; Intracerebroventricular (ICV) Enzyme Replacement Therapy
• 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
• 3) By Genistein: Oral Genistein Supplementation; High-Dose Genistein Regimens; Low-Dose Genistein Regimens
• 4) By Other Treatments: Substrate Reduction Therapy (SRT); Stem Cell Therapy; Anti-Inflammatory Agents; Symptomatic Management Drugs
• Companies Mentioned: Takeda Pharmaceutical Company Limited; BioMarin Pharmaceutical Inc.; Qiagen N.V.; Natera Inc.; Guardant Health Inc.
• Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
• 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
• Intravenous Enzyme Replacement Therapy
• Intrathecal Enzyme Replacement Therapy
• Intracerebroventricular (ICV) Enzyme Replacement Therapy
• 6.5. Global Sanfilippo Syndrome Market, Sub-Segmentation Of Gene Therapy, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• Adeno-Associated Virus (AAV)-Based In Vivo Gene Therapy
• Lentiviral Vector-Based Ex Vivo Gene Therapy
• Non-Viral Vector Gene Delivery
• 6.6. Global Sanfilippo Syndrome Market, Sub-Segmentation Of Genistein, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• Oral Genistein Supplementation
• High-Dose Genistein Regimens
• Low-Dose Genistein Regimens
• 6.7. Global Sanfilippo Syndrome Market, Sub-Segmentation Of Other Treatments, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• Substrate Reduction Therapy (SRT)
• Stem Cell Therapy
• Anti-Inflammatory Agents
• Symptomatic Management Drugs

7. Sanfilippo Syndrome Market Regional And Country Analysis

• 7.1. Global Sanfilippo Syndrome Market, Split By Region, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 7.2. Global Sanfilippo Syndrome Market, Split By Country, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

8. Asia-Pacific Sanfilippo Syndrome Market

• 8.1. Asia-Pacific Sanfilippo Syndrome Market Overview
• Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
• 8.2. Asia-Pacific Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 8.3. Asia-Pacific Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 8.4. Asia-Pacific Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

9. China Sanfilippo Syndrome Market

• 9.1. China Sanfilippo Syndrome Market Overview
• 9.2. China Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion
• 9.3. China Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion
• 9.4. China Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion

10. India Sanfilippo Syndrome Market

• 10.1. India Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 10.2. India Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 10.3. India Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

11. Japan Sanfilippo Syndrome Market

• 11.1. Japan Sanfilippo Syndrome Market Overview
• 11.2. Japan Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 11.3. Japan Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 11.4. Japan Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

12. Australia Sanfilippo Syndrome Market

• 12.1. Australia Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 12.2. Australia Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 12.3. Australia Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

13. Indonesia Sanfilippo Syndrome Market

• 13.1. Indonesia Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 13.2. Indonesia Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 13.3. Indonesia Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

14. South Korea Sanfilippo Syndrome Market

• 14.1. South Korea Sanfilippo Syndrome Market Overview
• 14.2. South Korea Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 14.3. South Korea Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 14.4. South Korea Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

15. Western Europe Sanfilippo Syndrome Market

• 15.1. Western Europe Sanfilippo Syndrome Market Overview
• 15.2. Western Europe Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 15.3. Western Europe Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 15.4. Western Europe Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

16. UK Sanfilippo Syndrome Market

• 16.1. UK Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 16.2. UK Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 16.3. UK Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

17. Germany Sanfilippo Syndrome Market

• 17.1. Germany Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 17.2. Germany Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 17.3. Germany Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

18. France Sanfilippo Syndrome Market

• 18.1. France Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 18.2. France Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 18.3. France Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

19. Italy Sanfilippo Syndrome Market

• 19.1. Italy Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 19.2. Italy Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 19.3. Italy Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

20. Spain Sanfilippo Syndrome Market

• 20.1. Spain Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 20.2. Spain Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 20.3. Spain Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

21. Eastern Europe Sanfilippo Syndrome Market

• 21.1. Eastern Europe Sanfilippo Syndrome Market Overview
• 21.2. Eastern Europe Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 21.3. Eastern Europe Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 21.4. Eastern Europe Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

22. Russia Sanfilippo Syndrome Market

• 22.1. Russia Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 22.2. Russia Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 22.3. Russia Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

23. North America Sanfilippo Syndrome Market

• 23.1. North America Sanfilippo Syndrome Market Overview
• 23.2. North America Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 23.3. North America Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 23.4. North America Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

24. USA Sanfilippo Syndrome Market

• 24.1. USA Sanfilippo Syndrome Market Overview
• 24.2. USA Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 24.3. USA Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 24.4. USA Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

25. Canada Sanfilippo Syndrome Market

• 25.1. Canada Sanfilippo Syndrome Market Overview
• 25.2. Canada Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 25.3. Canada Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 25.4. Canada Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

26. South America Sanfilippo Syndrome Market

• 26.1. South America Sanfilippo Syndrome Market Overview
• 26.2. South America Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 26.3. South America Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 26.4. South America Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

27. Brazil Sanfilippo Syndrome Market

• 27.1. Brazil Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 27.2. Brazil Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 27.3. Brazil Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

28. Middle East Sanfilippo Syndrome Market

• 28.1. Middle East Sanfilippo Syndrome Market Overview
• 28.2. Middle East Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 28.3. Middle East Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 28.4. Middle East Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

29. Africa Sanfilippo Syndrome Market

• 29.1. Africa Sanfilippo Syndrome Market Overview
• 29.2. Africa Sanfilippo Syndrome Market, Segmentation By Treatment, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 29.3. Africa Sanfilippo Syndrome Market, Segmentation By Diagnosis, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
• 29.4. Africa Sanfilippo Syndrome Market, Segmentation By End-User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

30. Sanfilippo Syndrome Market Competitive Landscape And Company Profiles

• 30.1. Sanfilippo Syndrome Market Competitive Landscape
• 30.2. Sanfilippo Syndrome Market Company Profiles
  • 30.2.1. Takeda Pharmaceutical Company Limited Overview, Products and Services, Strategy and Financial Analysis
  • 30.2.2. BioMarin Pharmaceutical Inc. Overview, Products and Services, Strategy and Financial Analysis
  • 30.2.3. Qiagen N.V. Overview, Products and Services, Strategy and Financial Analysis
  • 30.2.4. Natera Inc. Overview, Products and Services, Strategy and Financial Analysis
  • 30.2.5. Guardant Health Inc. Overview, Products and Services, Strategy and Financial Analysis

31. Sanfilippo Syndrome Market Other Major And Innovative Companies

• 31.1. Novogene Corporation
• 31.2. Ultragenyx Pharmaceutical Inc.
• 31.3. JCR Pharmaceuticals Co. Ltd.
• 31.4. Oxford Nanopore Technologies plc
• 31.5. Pacific Biosciences of California Inc.
• 31.6. Denali Therapeutics Inc.
• 31.7. Macrogen Inc.
• 31.8. REGENXBIO Inc.
• 31.9. Personalis Inc.
• 31.10. Veritas Genetics Inc.
• 31.11. Greenwood Genetic Center
• 31.12. ArmaGen Inc.
• 31.13. Abeona Therapeutics Inc.
• 31.14. Orchard Therapeutics plc
• 31.15. Allievex Corporation

32. Global Sanfilippo Syndrome Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Sanfilippo Syndrome Market

34. Recent Developments In The Sanfilippo Syndrome Market

35. Sanfilippo Syndrome Market High Potential Countries, Segments and Strategies

• 35.1 Sanfilippo Syndrome Market In 2029 - Countries Offering Most New Opportunities
• 35.2 Sanfilippo Syndrome Market In 2029 - Segments Offering Most New Opportunities
• 35.3 Sanfilippo Syndrome Market In 2029 - Growth Strategies
  • 35.3.1 Market Trend Based Strategies
  • 35.3.2 Competitor Strategies

36. Appendix

• 36.1. Abbreviations
• 36.2. Currencies
• 36.3. Historic And Forecast Inflation Rates
• 36.4. Research Inquiries
• 36.5. The Business Research Company
• 36.6. Copyright And Disclaimer
• 250617_r34959_Sanfilippo_Syndrome_GMR_2025