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Global hunter syndrome treatment market trends and forecast to 2030

DelveInsight's Hunter Syndrome Market Insights, Epidemiology, and Market Forecast-2030<br><br>https://www.delveinsight.com/report-store/hunter-syndrome-market

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Global hunter syndrome treatment market trends and forecast to 2030

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  1. 2020 Hunter Hunter Syndrome Syndrome Market Insights, Epidem Market Insights, Epidemiology, and Market Forecast Forecast- - 2030 2030 iology, and Market

  2. D e l v e I n s i g h t | P a g e | 1 1 Key Insights The Report contains: The Report contains: Pipeline analysis across different stages of development (Phase III), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross- competition, launch date along with product activities Overview of pathophysiology, various diagnostic approaches and treatment including detailed chapters for marketed products emerging therapies Research Research Pipeline Pipeline Research Research Analysis Analysis Analysis Analysis Analysis Analysis Research Research Research Research Analysis Analysis Pipeline Pipeline Valuation Valuation Valuation Valuation Valuation Valuation Pipeline Pipeline Pipeline Pipeline Valuation Valuation algorithm and development Epidemiology Epidemiology Forecast Forecast Forecast Forecast Forecast Forecast Epidemiology Epidemiology Epidemiology Epidemiology Market Market Market Market Market Market Market Market Analysis Analysis Analysis Analysis Analysis Analysis Analysis Analysis Historical as well as forecasted epidemiology in the United States, EU5, and Japan from 2017 to 2030 Detailed forecasted market covering the United States, EU5, and Japan, from 2017 to 2030 historical and Market size by Market size by therapies therapies Market size by Market size by therapies therapies Detailed Market size by therapies, covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, from 2017 to 2030 Strategic analysis of Treatment options & Emerging Therapies Insightful Reviews on Market Drivers & Market Barriers Healthcare Market Research DelveInsight Copyright

  3. D e l v e I n s i g h t | P a g e | 2 2 DelveInsight’s “Hunter Syndrome Hunter Syndrome - - Market Insights, Epidemiology & Market Insights, Epidemiology & Market Market Forecast Forecast- -20 provides an overview of the disease and market size of Hunter Syndrome for the seven major pharmaceutical markets i.e., the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. This report covers the various treatment practices, and Hunter Syndrome forecasted epidemiology from 2020 to 2030, segmented by the seven major markets. Key Strengths Key Strengths ❖1 11 1 Year Forecast Year Forecast Report Introduction Report Introduction 2030 30” report The report provides an understanding of the epidemiology and changing market dynamics of Hunter Syndrome in the seven major pharmaceutical markets for the period 2017–2030 explaining its causes and currently available therapies. ✓Comprehensive insight has been provided into the epidemiology of the Hunter Syndrome and its treatment in the 7MM. ✓Additionally, an all-inclusive account of both the current and emerging therapies for Hunter Syndrome is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape. ✓A detailed review of historical and forecasted Hunter Syndrome market is included in the report, covering drug outreach in the 7MM. ✓The report provides an edge while developing business strategies, by understanding trends shaping and driving the Hunter Syndrome market. ✓The report covers the descriptive overview of Hunter Syndrome, ❖7MM Coverage 7MM Coverage ❖Epidemiology Segmentation Epidemiology Segmentation ❖Drugs Uptake Drugs Uptake ❖Highly Analyzed Market Highly Analyzed Market ❖Key Cross Key Cross Competition Competition ❖Market Size by Country Market Size by Country ❖Market Size by Therapy Market Size by Therapy Key Assessments Key Assessments ❖Current Treatment Practices Current Treatment Practices ❖Unmet Needs Unmet Needs ❖Emerging Drug Profiles Emerging Drug Profiles ❖Market Attractiveness Market Attractiveness Healthcare Market Research DelveInsight Copyright

  4. D e l v e I n s i g h t | P a g e | 3 3 Epidemiology Epidemiology Insights Insights Hunter Hunter Syn sulfatase deficiency. sulfatase deficiency. It is an inherited disorder of carbohydrate metabolism that occurs almost exclusively in males in males. MPS II is caused by mutations in the IDS gene and is inherited in an X-linked manner. Females are generally unaffected carriers of this condition. Syndrome, also known as Mucopolysaccharidosis type II (MPS I drome, also known as Mucopolysaccharidosis type II (MPS II I), ), I2S deficiency I2S deficiency and Iduronate 2 almost exclusively Iduronate 2- - As per N National medicine in Manitoba, Canada, who first described two brothers with the disease in 1917. MPS II has a wide range of symptoms that vary in severity and can be managed and treated with enzyme replacement therapies. There is no cure for MPS II and it affects 1 in 100,000 to 1 in 170,000 primarily males. ational MPS Society MPS Society, Hunter syndrome takes its name from Charles Hunter, the professor of It is characterized by distinctive facial features, a large head, hydrocephalus, enlargement of the liver and spleen (hepatosplenomegaly), umbilical or inguinal hernia, and hearing loss. Individuals with this condition may additionally have joint deformities and heart abnormalities involving the valves. At birth, individuals with MPS II do not display any features of the condition. Between ages 2 and 4, they develop full lips, large rounded cheeks, a broad nose, and an enlarged tongue (macroglossia). The vocal cords also gets enlarged, which results in a deep, hoarse voice. Narrowing of the airway causes frequent upper respiratory infections and short pauses in breathing during sleep (sleep apnea). As the disorder progresses, individuals need medical assistance to keep their airway open. Hunter Hunter Syndrome Syndrome – –Epidemiology Epidemiology by Gender by Gender •According to the National Organization for Rare Disorders National Organization for Rare Disorders, MPS II occurs in approximately 1 in 100,000 to 1 in 170,000 male births. •As per the study published by Maurizio Scarpa et al., Maurizio Scarpa et al., titled, “Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease” recommendations for the diagnosis and multidisciplinary management of a rare disease” MPS II is one of the most common mucopolysaccharidoses, with an estimated prevalence of 1 in 140,000-156,000 live births in Europe. •According to the study by Shaukat Khan et al., Shaukat Khan et al., “ “In Japan, the highest birth prevalence was 0.84 for MPS II, accounting for 55% of all MPS. MPS I, III, and IV accounted for 15, 16, and 10%, respectively.” •Recent studies from Germany and the Netherlands report an overall incidence of 1 case in 140,000-330,000 live births, and, more specifically, 1 case per 77,000 male births. “Mucopolysaccharidosis type II: European Healthcare Market Research DelveInsight Copyright

  5. D e l v e I n s i g h t | P a g e | 4 4 Epidemiology Segmentation-7MM Total Prevalent Cases of Total Prevalent Cases of Hunter ( (2017 2017– –2030 2030) ) Hunter Syndrome Syndrome 1 1 Severity Severity Specific Severe Form] Severe Form] ( (2017 Specific Prevalence of Prevalence of Hunter 2017– –2030 2030) ) Hunter Syndrome Syndrome [Mild and [Mild and 2 2 2 2 Diagnosed and Treatable Population of Diagnosed and Treatable Population of Hunter ( (2017 2017– –2030 2030) ) Hunter Syndrome Syndrome 3 3 There are two types of MPS II, called the severe and mild types mild types. While both types affect many different organs and tissues as described above, people with severe MPS II also experience a decline in intellectual severe MPS II also experience a decline in intellectual function and a more rapid disease progression. function and a more rapid disease progression. Individuals with the severe form begin to lose basic functional skills (developmentally regress) between the ages of 6 and 8. The life expectancy of these individuals is 10-20 years. Individuals with mild MPS II mild MPS II also have a shortened lifespan, but they typically live they typically liveinto adulthood and their intelligence is not affected. intelligence is not affected. Heart disease and airway obstruction are major causes of death in people with both types of MPS II. severe and Severity specific prevalence of Hunter Syndrome people with into adulthood and their Mild Form Severe Form Healthcare Market Research DelveInsight Copyright

  6. D e l v e I n s i g h t | P a g e | 5 5 Graphical Representation of Graphical Representation of Hunter Total Prevalent Cases Total Prevalent Cases of Hunter Syndrome Syndrome Epidemiology Epidemiology of Hunter Hunter Syndrome Syndrome in in the the 7MM 7MM ( (2017 2017– –2030 2030) ) Total Prevalent Cases in 7MM Population in Numbers (N) 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 United States France Italy Spain Germany United Kingdom Japan Diagnosed and Treatable Diagnosed and Treatable Cases of Cases of Hunter Syndrome Hunter Syndrome in 7MM in 7MM (2017 (2017– –2030 2030) ) Diagnosed and Treatable Cases Population in Numbers (N) 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 Diagnosed Cases Treated Cases Healthcare Market Research DelveInsight Copyright

  7. D e l v e I n s i g h t | P a g e | 6 6 Marke Market Insights t Insights Market Outlook: Hunter Syndrome The treatment of MPS II disease is multi-disciplinary and is best treated by a team of specialists, who have knowledge about the disease, and who can offer supportive and symptomatic care. Care is directed toward relieving the symptoms of the disease. Enzyme replacement therapy (ERT) Enzyme replacement therapy (ERT) is now available for MPS II and this treatment ameliorates the somatic features of the disorder but does not treat the central nervous system. The ERT listed below has been approved 2006 2006 for the treatment of this condition: approved by the Food and Food and Drug Drug Administration Administration (FDA) as orphan orphan products products in •Idursulfase Idursulfase (Brand name: Elaprase) (Brand name: Elaprase) – – It is sponsored by Shire the original developer original developer of the drug, TKT Therapeutics, FDA FDA- -approved indication: approved indication: Hunter syndrome (mucopolysaccharidosis II, MPS II). Idursulfase shown to improve walking capacity in these patients shown to improve walking capacity in these patients. . Idursulfase is still in phase II/III phase II/III for Mucopolysaccharidosis II (in adolescents, in children) in Spain, USA, Argentina, Mexico, United Kingdom, Australia, Canada, Colombia and France. Shire (now Takeda) (now Takeda), a company that acquired TKT Therapeutics, in 2005. acquired Idursulfase has been has been Other t Other treatments of MPS II reatments of MPS II are symptomatic and supportive. are symptomatic and supportive. These management interventions commonly include developmental, occupational developmental, occupational, , and physical therapy and physical therapy. Hernias and joint contractures may be corrected by surgery surgery. Tonsillectomy and adenoidectomy Tonsillectomy and adenoidectomy (surgical removal of both the tonsils and the adenoids) can be used to alleviate breathing and swallowing problems. Positive pressure ventilation or tracheostomy) can be used to apply mild air pressure to keep the airway open for proper breathing. C Carpal tunnel release arpal tunnel release can treat carpal tunnel syndrome. Surgical implantation be used to treat possible hydrocephalus. Hearing devices Hearing devices may be prescribed to treat hearing loss. Developmental, physical, and occupational therapy can be helpful. Genetic counseling is recommended for families that have a child with MPS II. Positive pressure ventilation (CPAP Surgical implantation of a ventricular shunt ventricular shunt may Alternatives such as stem cell transplantation (STC) using the umbilical cord blood, peripheral blood hematopoietic cells or bone marrow have also been used, but they appear to offer limited clinical benefits in patients with this disease and have been associated with a serious risk of morbidity and mortality. Though enzyme replacement therapy by idursulfase may improve certain symptoms, it does not cross the blood- brain barrier (BBB) and will not alleviate neurological symptoms. Therefore, there is a significant unmet need need for a more efficacious therapy which is capable of crossing BBB. significant unmet To overcome these challenges To overcome these challenges researchers have focused on the development of well-tolerated therapies that can cross the blood-brain barrier. One approach that has been explored is the infusion of ERT into the cerebrospinal fluid, thereby enabling widespread distribution throughout the CNS. Other areas of research include the use of phar pharmacological chaperones, gene therapy macological chaperones, gene therapy, , and substrate reduction therapy. unmet needs include lack of curative treatment for MPS II, lack of accurate diagnostic tests, etc. and substrate reduction therapy. Also, the Healthcare Market Research DelveInsight Copyright

  8. D e l v e I n s i g h t | P a g e | 7 7 The Hunter Syndrome market is expected to experience a positive growth in the coming years owing to the already prescribed product along with the launch of distinctive emerging therapies in the coming years. Additionally, due to the lack of therapies which effectively stop or slow the disease progression a potential opportunity exists for the development and launch of novel therapies. The market size of Hunter syndrome is expected to increase at a significant growth during the study period [2017–2030] Current treatment in Hunter syndrome landscape has only one approved Enzyme Replacement Therapy, Stem Cell Transplantation and other off-label therapies Expected launch of JR-141 (JCR Pharmaceuticals) and many others for treating Hunter syndrome shall fuel the growth of the market Many clinical trials are evaluating potential treatments enrolling people with Hunter syndrome. Hence, increasing R&D activities, and positive results of these trials shall fuel the therapeutic market size of Hunter syndrome The therapies under development are focused on novel approaches to treat/improve the disease condition Region-wise Market Size of Hunter Syndrome (2017–2030) 2017 2030 US US DE DE FR FR IT IT ESP ESP UK UK JP JP Healthcare Market Research DelveInsight Copyright

  9. D e l v e I n s i g h t | P a g e | 8 8 Graphical Representation of Graphical Representation of Hunter Hunter Syndrome Syndrome Market Size Market Size in 7MM in 7MM Total Total Market Size of Market Size of Hunter Hunter Syndrome Syndrome in the 7MM in the 7MM ( (2017 2017– –2030 2030) ) Total Market Size of Hunter Syndrome (INDICATIVE) USD, Million 2017 2018 2019 2020 UK 2021 Germany 2022 2023 2024 2025 Spain 2026 2027 2028 Japan 2029 2030 USA France Italy Market Size of Market Size of Hunter Hunter Syndrome Syndrome in the U in the United nited S State tates s ( (2017 2017– –2030 2030) ) Market Size of Hunter Syndrome by Upcoming Therapies (INDICATIVE) 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 Therapy 1 Therapy 2 Therapy 3 Therapy 4 Healthcare Market Research DelveInsight Copyright

  10. D e l v e I n s i g h t | P a g e | 9 9 Drug Uptak Drug Uptake e Emerging Drugs Analysis The dynamics of Hunter syndrome market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the disease, incremental healthcare spending across the world, and expected the launch of emerging therapies during the forecast period of 2020–2030. Companies like JCR Pharmaceuticals JCR Pharmaceuticals ( (JR JR- -141 Hunterase), Regenxbio Inc. (RGX Hunterase), Regenxbio Inc. (RGX- -121) 121), , and others and others are working toward the development of new treatment therapies for Hunter syndrome. While some of the companies have recently shifted their focus toward this indication, whereas others are expected to create a significant influence on the market size during the forecast period. 141) ), Green Cross Corporation (GC1111B or , Green Cross Corporation (GC1111B or JR JR- -141 141 (JCR Pharmaceuticals) is a blood-brain-barrier penetrating therapeutic enzyme for Hunter syndrome currently undergoing one Phase III (Japan) and one Phase II (Brazil) clinical trials. In March 2018, JR-141 was designated under the SAKIGAKE Designation System SAKIGAKE Designation System in Japan by the Ministry of Health, Labour and Welfare, and is planning to apply for marketing approval in 2020. It has also received Orphan Drug Designation from both the EMA and the FDA. GC1111B or Hunterase GC1111B or Hunterase developed by Green Cross is the world’s second Hunter syndrome drug (recombinant iduronate-2-sulfatase) and is a biobetter of Shire’s Elaprase The USFDA had also awarded Green Cross, the official Orphan Drug Designation 2013. It has already been approved in South Korea and marketed in eight countries in South America and North Africa. It is currently undergoing a phase II study to determine the safety and efficacy in the United States. However, there is another Phase III clinical trial going on in Korea. If once approved, it will open a new paradigm for patients suffering from Hunter Syndrome, offering a distinct therapeutic alternative with improved efficacy and safety. Shire’s Elaprase used to treat Hunter syndrome. Orphan Drug Designation for Hunterase back in Apart from these, Denali Therap recombinant enzyme engineered to cross the blood-brain barrier (Denali’s Enzyme Transport Vehicle (ETV) Technology) and to replace the Iduronate enzyme and treat neuropathic and systemic forms of the disease. The company will start its Phase I/II trial by June 2020. However, pre-clinical studies with DNL310 in a relevant disease model demonstrated increased brain uptake and a more robust reduction of downstream markers of disease in the central nervous system compared to standard ERT. It has also been granted orphan drug product orphan drug product and rare pediatric disease rare pediatric disease designation designation by the USFDA. Denali Therapeutics’ eutics’ is developing DNL310 DNL310 (Iduronate 2-sulfatase) which is a Additionally, there are many other potential therapies results during the early phase clinical trials. For example, Sangamo Therapeutics Drug Designation Drug Designation and Fast Track designation Fast Track designation from the FDA for SB Treatment for MPS II. RGX RGX- -121 121 (Regenxbio) (Regenxbio) has received orphan drug product, rare pediatric disease Fast Track designation from the FDA. Fast Track designation from the FDA. potential therapies under trial for MPS II which have shown promising Sangamo Therapeutics has received an Orphan SB- -913 ( 913 (Phase I/II) orphan drug product, rare pediatric disease, , and Orphan Phase I/II) as a Genome Editing Healthcare Market Research DelveInsight Copyright

  11. D e l v e I n s i g h t | P a g e | 10 10 Table of Content to View 1.Report Introduction Report Introduction 2.Hunter Hunter S Syndrome yndrome Market Overview at a Glance Market Overview at a Glance 2.1.7 Major Market Size (%) of Hunter syndrome in 2017 2.2.7 Major Market Size (%) of Hunter syndrome in 2030 3.Disease Background and Overview: Disease Background and Overview: Hunter 3.1.Introduction 3.2.Causes and Risk Factors 3.3.Pathophysiology 3.4.Signs and Symptoms 3.5.Diagnosis 4.Epidemiology and Patient Population Epidemiology and Patient Population 4.1.Key Findings 4.2.Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] 4.3.Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] 4.4.Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] 5.Epidemiology of Epidemiology of Hunter Hunter syndrome syndrome by Countries ( by Countries (2017 5.1.United States United States- - Epidemiology ( Epidemiology (2017 2017– –2030 5.1.1.Assumptions and Rationale 5.1.2.Total Prevalent Population of Hunter syndrome in the US [2017–2030] 5.1.3.Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] 5.1.4.Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] 5.2.EU EU- -5 5 - - Epidemiology ( Epidemiology (2017 2017– –203 2030 0) ) 5.2.1.Assumptions and Rationale 5.3.Germany Germany- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.3.1.Total Prevalent Population of Hunter syndrome in Germany [2017–2030] 5.3.2.Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] 5.3.3.Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] 5.4.France France- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.4.1.Total Prevalent Population of Hunter syndrome in France [2017–2030] 5.4.2.Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] 5.4.3.Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] 5.5.Italy Italy- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.5.1.Total Prevalent Population of Hunter syndrome in Italy [2017–2030] 5.5.2.Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] 5.5.3.Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] 5.6.Spain Spain- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.6.1.Total Prevalent Population of Hunter syndrome in Spain [2017–2030] 5.6.2.Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030] Hunter syndrome syndrome 2017– –2030 2030) ) 2030) ) Healthcare Market Research DelveInsight Copyright

  12. D e l v e I n s i g h t | P a g e | 11 11 5.6.3.Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] 5.7.United Kingdom United Kingdom- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.7.1.Total Prevalent Population of Hunter syndrome in the UK [2017–2030] 5.7.2.Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] 5.7.3.Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] 5.8.Japan Japan- -Epidemiology ( Epidemiology (2017 2017– –2030 2030) ) 5.8.1.Total Prevalent Population of Hunter syndrome in Japan [2017–2030] 5.8.2.Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] 5.8.3.Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] 6.Treatments & Medical Practices Treatments & Medical Practices 6.1.Treatment Algorithm 6.2.Treatment Guidelines 7.Marketed Products Marketed Products 7.1.Elaprase: Shire Elaprase: Shire 7.1.1.Drug description 7.1.2.Regulatory milestones 7.1.3.Generic Availability 7.1.4.Safety and efficacy 7.1.5.Product Profile 8.Emerging Therapies Emerging Therapies 8.1.Key Cross Competition Key Cross Competition 8.2. JR JR- -141: JCR Pharmaceuticals 141: JCR Pharmaceuticals 8.2.1.Product Description 8.2.2.Regulatory Milestones 8.2.3.Clinical Development 8.2.4.Clinical Pipeline Activity 8.2.5.Ongoing Trials Information 8.2.6.Safety and efficacy 8.2.7.Product Profile 8.3.GC1111B or Hunterase: Green Cross Corporation GC1111B or Hunterase: Green Cross Corporation 8.3.1.Product Description 8.3.2.Regulatory Milestones 8.3.3.Clinical Development 8.3.4.Clinical Pipeline Activity 8.3.5.Ongoing Trials Information 8.3.6.Safety and efficacy 8.3.7.Product Profile 8.4.RGX RGX- -121: Regenxbio Inc. 121: Regenxbio Inc. 8.4.1.Product Description 8.4.2.Regulatory Milestones Healthcare Market Research DelveInsight Copyright

  13. D e l v e I n s i g h t | P a g e | 12 12 8.4.3.Clinical Development 8.4.4.Clinical Pipeline Activity 8.4.5.Ongoing Trials Information 8.4.6.Safety and efficacy 8.4.7.Product Profile List to be continued in report… 9.Hunter Hunter syndrome syndrome Market Size 9.1.Key Findings 9.2.Total 7MM Hunter syndrome Market Analysis 9.3.Overview of Total Hunter syndrome Market 9.4.Market size of Hunter syndrome Market by 7MM (2017–2030) 10.7MM: 7MM: Country Country- -Wise Market Analysis Wise Market Analysis 10.1.United States Market Size United States Market Size 10.1.1.Total Market Size of Hunter syndrome 10.1.2.Market Size of Hunter syndrome by Therapies 10.2.Germany Market Size Germany Market Size 10.2.1.Total Market Size of Hunter syndrome 10.2.2.Market Size of Hunter syndrome by Therapies 10.3.France France Market Size Market Size 10.3.1.Total Market Size of Hunter syndrome 10.3.2.Market Size of Hunter syndrome by Therapies 10.4.Italy Market Size Italy Market Size 10.4.1.Total Market Size of Hunter syndrome 10.4.2.Market Size of Hunter syndrome by Therapies 10.5.Spain Market Size Spain Market Size 10.5.1.Total Market Size of Hunter syndrome 10.5.2.Market Size of Hunter syndrome by Therapies 10.6.United Kingdom Market Size United Kingdom Market Size 10.6.1.Total Market Size of Hunter syndrome 10.6.2.Market Size of Hunter syndrome by Therapies 10.7.Japan Market Size Japan Market Size 10.7.1.Total Market Size of Hunter syndrome 10.7.2.Market Size of Hunter syndrome by Therapies 11.Market Drivers Market Drivers 12.Market Barriers Market Barriers 13.Report Methodology Report Methodology Market Size Healthcare Market Research DelveInsight Copyright

  14. D e l v e I n s i g h t | P a g e | 13 13 13.1.Sources Used 14.DelveInsight Capabilities DelveInsight Capabilities 15.Disclaimer Disclaimer 16.About DelveInsight About DelveInsight * *Table of Contents is not exhaustiv Table of Contents is not exhaustive e; will be provided in the final ; will be provided in the final report report Healthcare Market Research DelveInsight Copyright

  15. D e l v e I n s i g h t | P a g e | 14 14 List of Tables Table 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] Table 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] Table 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] Table 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030] Table 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] Table 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] Table 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030] Table 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] Table 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] Table 10 Total Prevalent Population of Hunter syndrome in France [2017–2030] Table 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] Table 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] Table 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030] Table 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] Table 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] Table 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030] Table 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030] Table 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] Table 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030] Table 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] Table 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] Table 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030] Table 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] Table 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] Table 25 JR-141, Clinical Trials by Recruitment status, 2020 Table 26 JR-141, Clinical Trials by Zone, 2020 Table 27 GC1111B, Clinical Trials by Recruitment status, 2020 Table 28 GC1111B, Clinical Trials by Zone, 2020 Table 29 RGX-121, Clinical Trials by Recruitment status, 2020 Table 30 RGX-121, Clinical Trials by Zone, 2020 Table 31 Market size of Hunter syndrome Market by 7MM (2017–2030) Table 32 Region-wise Market size of Hunter syndrome, USD Million (2017–2030) Table 33 7MM-Market Size by Therapy in USD, Million (2017–2030) Table 34 United States Market Size of Hunter syndrome in USD, Million (2017–2030) Table 35 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 36 Germany Market Size of Hunter syndrome in USD, Million (2017–2030) Table 37 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 38 France Market Size of Hunter syndrome in USD, Million (2017–2030) Table 39 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 40 Italy Market Size of Hunter syndrome in USD, Million (2017–2030) Healthcare Market Research DelveInsight Copyright

  16. D e l v e I n s i g h t | P a g e | 15 15 Table 41 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 42 Spain Market Size of Hunter syndrome in USD, Million (2017–2030) Table 43 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 44 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030) Table 45 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 46 Japan Market Size of Hunter syndrome in USD, Million (2017–2030) Table 47 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) * *List of Table is not exhaustive List of Table is not exhaustive; will be provided in the final report ; will be provided in the final report Healthcare Market Research DelveInsight Copyright

  17. D e l v e I n s i g h t | P a g e | 16 16 List of Figures Figure 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] Figure 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] Figure 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] Figure 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030] Figure 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] Figure 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] Figure 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030] Figure 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] Figure 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] Figure 10 Total Prevalent Population of Hunter syndrome in France [2017–2030] Figure 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] Figure 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] Figure 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030] Figure 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] Figure 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] Figure 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030] Figure 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030] Figure 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] Figure 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030] Figure 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] Figure 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] Figure 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030] Figure 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] Figure 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] Figure 25 Unmet Needs Figure 26 Market size of Hunter syndrome Market by 7MM (2017–2030) Figure 27 Region-wise Market size of Hunter syndrome, USD Million (2017–2030) Figure 28 7MM-Market Size by Therapy in USD, Million (2017–2030) Figure 29 United States Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 30 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 31 Germany Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 32 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 33 France Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 34 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 35 Italy Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 36 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 37 Spain Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 38 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 39 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 40 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Healthcare Market Research DelveInsight Copyright

  18. D e l v e I n s i g h t | P a g e | 17 17 Figure 41 Japan Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 42 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 43 Market Drivers Figure 44 Market Barriers *List of Figures is not List of Figures is not exhaustive exhaustive; will be provide in the final report ; will be provide in the final report Healthcare Market Research DelveInsight Copyright

  19. D e l v e I n s i g h t | P a g e | 18 18 Appendix Report Methodology DelveInsight is a premier source for pharmaceutical and biotechnology sector analysis, delivering exclusive, trusted commercial insight into industry performance through its proprietary platform. DelveInsight’s reports are continuously updated and revised. The following research methodology is followed for all databases and reports. Coverage Coverage The objective of updating DelveInsight coverage is to ensure that it represents the most up-to- date vision of the industry possible. The DelveInsight is a fully integrated solution for comprehensive intelligence on various pharmaceutical products, both in the market and in the pipeline, across the globe. Research Design Research Design Secondary Research Secondary Research The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market. The secondary research sources that are typically referred to include, but are not limited to: •Company websites, annual reports, financial reports, broker reports, investor presentations, and SEC Filings Industry trade journals and other literature. •Internal and external proprietary databases. •National government documents, statistical databases, and market reports. •News articles, press releases, and web-casts specific to the companies operating in the market. Primary Research Primary Research DelveInsight conducts primary research by interacting with the healthcare professionals, doctors, industry’s experts, and industry participants and commentators in order to validate its data and analysis. The discussion provides first-hand information on the market size, market trends, growth trends, competitive landscape, and future outlook, for example. •It helps in validating and strengthening the secondary research findings. •It further develops the Analysis Team’s expertise and market understanding. •An interactive one-stop, fully integrated business intelligence (BI) platform, catering the need of the global pharmaceuticals market, across all the value chains. •The comprehensive integration allows for the seamless comparison of the market dynamics, helps strategic planning, and decision making to stay competitive in the global pharmaceuticals industry. •We help our clients to find answers relevant to business decision-making, including potential market opportunities, competitor assessments, and business environmental dynamics enhances companies to plan development, trials or process-based research on their competitor focus. Healthcare Market Research DelveInsight Copyright

  20. D e l v e I n s i g h t | P a g e | 19 19 •Fo •DelveInsight Reports are prepared considering different epidemiology and market factors, keeping past and current trends in focus. Our forecasting models are prepared based on the Hybrid approach, where we use both top to bottom and bottom to top approaches and analyze different factors and parameters to forecast the numbers. • •We develop a deep understanding by doing systemic literature research for Epidemiological data around different geographies. We prepare the forecast model based on different data sources, considering past and current trends from different factors such as improvements in survival and disease therapy, unmet need, the risk of disease by gender and age group, population size, diagnose trends, treatment factors, and others. • •Our Market Forecast model consider factors such as unmet medical need, drug competitor factor, patient compliance, product price, marketed drugs calibration based on actual sales and their impact in different geographies, emerging drugs and their impact, novel agents analysis based on their results, therapeutic potential, regulatory success, launch dates, etc., market challenges, drivers and barriers, and other factors. • •Our major sources are company annual reports, scientific literature, epidemiology studies conducted by different agencies, Pub-Med Articles, the country-specific government released population sources, and others. To keep up with current market trends, we try to take doctors, KOLs, and industry experts’ opinion through secondary as well as primary research to come up with the actual market size. • •Expert Panel Validation Expert Panel Validation •DelveInsight uses a panel of experts to cross verify research and forecast methodologies and drive its analytical content. The panel comprises of marketing managers, product specialists, international sales managers from pharmaceutical companies; academics from research universities and consultants from professional services companies. Forecast Methodology recast Methodology Healthcare Market Research DelveInsight Copyright

  21. D e l v e I n s i g h t | P a g e | 20 20 DelveInsight Capabilities Our Consulting Services collaborate with Biotech & Pharmaceutical companies, Venture Capitalist, R&D units, and API manufacturers to help them in critical business decisions, building commercial excellence and growing businesses in an increasingly competitive and challenging environment. Competitive Intelligence Conference/Congress Intelligence Market/BD Assessment Studies R&D Analytics Forecasting Primary CI 1 2 3 4 DelveInsight delivers strategic CI Inputs to clients for monitoring competition and staying ahead via our expertise in gathering industry intelligence, leading to better decision-making. Our in-depth insights help market research and business development teams of global bio-Pharma companies in understanding the market, identifying and targeting the untapped opportunities. DelveInsight’s forecasting through epidemiology- and unit-based methodologies help create flexible and robust models to accommodate key inputs for effective market forecasting. DelveInsight’s CI team has supported many global clients by delivering CI projects with high-quality insights, helping improve their business processes, combining primary and secondary research methods and validating information to provide un-biased insights. 5 Our subject matter experts help deliver high-quality insights, supporting clients in key decisions like prioritizing indications, analyzing clinical trials, and analyzing the patents and publications. 6 Our in-house conference/congress intelligence experts gather information from various websites of congresses and analyze the data to map the KOLs attending the congresses, for effective planning and providing an un-biased analysis. Healthcare Market Research DelveInsight Copyright

  22. D e l v e I n s i g h t | P a g e | 21 21 Disclaimer All Rights Reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher, DelveInsight. The facts of this report were believed to be correct at the time of publication but cannot be guaranteed. Please note that the findings, conclusions, and recommendations that DelveInsight delivers will be based on information gathered in good faith from both primary and secondary sources, whose accuracy we were not always in a position to guarantee. As such DelveInsight can accept no liability whatsoever for the actions taken based on any information that may subsequently prove to be incorrect. About DelveInsight DelveInsight is a Business Consultant company and serves as a Knowledge partner across the value chain of the Pharmaceutical Industry. With the use of proprietary databases and analytical models, DelveInsight provides cutting-edge market and pipeline analysis and API intelligence across all therapy areas to the Pharma and biotech sector, helping clients to quantify market events and evaluate their impact on the valuation of products, portfolios, and companies. Additional information is available at www.delveinsight.com. For further information or queries For further information or queries, please contact the Sales Team, DelveInsight at salessupport@delveinsight.com or info@delveinsight.com Healthcare Market Research DelveInsight Copyright

  23. D e l v e I n s i g h t | P a g e | 22 22 REPORT PRICE Hunter Syndrome Hunter Syndrome Market Insights, Epidemiology Market Insights, Epidemiology, , and Market Forecast and Market Forecast- -20 2030 30 USD 6950 Single User License USD 10450 2-3 Users License USD 13900 Site License USD 20850 Corporate License Healthcare Market Research DelveInsight Copyright

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