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Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018
Summary
This report provides an overview of the pipeline landscape for mucopolysaccharidosis disorders, a group of inherited lysosomal storage disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for mucopolysaccharidosis I (MPS I) (Hurler syndrome), mucopolysaccharidosis II (MPS II) (Hunter syndrome) and mucopolysaccharidosis III (MPS III) (Sanfilippo syndrome), and features dormant and discontinued products.
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MPS I is caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 18 products in development for this indication.
MPS II is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 15 products in development for this indication.
MPS III is caused by an absence or malfunctioning of GAGs. Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). There are 18 products in development for this indication.
Companies operating in the mucopolysaccharidosis disorders pipeline space include ArmaGen, Sangamo and AngioChem.
Scope
- Which companies are the most active within each pipeline?
- Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
- To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
- What are the most important R&D milestones and data publications to have happened in this disease area?
Get Complete TOC With Tables and Figures @ https://www.researchmoz.us/mucopolysaccharidosis-disorders-drug-development-pipeline-review-2018-report.html/toc
Reasons to buy
- Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
- Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication and a comprehensive picture of recent updates and milestones for each
- Analyze the companies, institutions and universities currently operating in the pipeline and the products being fielded by each of these
- Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 5
1.2 List of Figures 6
2 Introduction 8
2.1 Mucopolysaccharidosis Disorders Report Coverage 8
2.2 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 8
2.3 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Overview 8
2.4 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview 8
3 Therapeutics Development 9
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3.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9
3.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14
3.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19
4 Therapeutics Assessment 23
4.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23
4.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28
4.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34
5 Companies Involved in Therapeutics Development 40
5.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 40
5.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 44
5.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 49
Continue...
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Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018
Summary
This report provides an overview of the pipeline landscape for mucopolysaccharidosis disorders, a group of inherited lysosomal storage disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for mucopolysaccharidosis I (MPS I) (Hurler syndrome), mucopolysaccharidosis II (MPS II) (Hunter syndrome) and mucopolysaccharidosis III (MPS III) (Sanfilippo syndrome), and features dormant and discontinued products.
To Get Sample Copy of Report visit @ https://www.researchmoz.us/enquiry.php?type=S&repid=1877887
MPS I is caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 18 products in development for this indication.
MPS II is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 15 products in development for this indication.
MPS III is caused by an absence or malfunctioning of GAGs. Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). There are 18 products in development for this indication.
Companies operating in the mucopolysaccharidosis disorders pipeline space include ArmaGen, Sangamo and AngioChem.
Scope
- Which companies are the most active within each pipeline?
- Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
- To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
- What are the most important R&D milestones and data publications to have happened in this disease area?
Get Complete TOC With Tables and Figures @ https://www.researchmoz.us/mucopolysaccharidosis-disorders-drug-development-pipeline-review-2018-report.html/toc
Reasons to buy
- Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
- Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication and a comprehensive picture of recent updates and milestones for each
- Analyze the companies, institutions and universities currently operating in the pipeline and the products being fielded by each of these
- Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 5
1.2 List of Figures 6
2 Introduction 8
2.1 Mucopolysaccharidosis Disorders Report Coverage 8
2.2 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 8
2.3 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Overview 8
2.4 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview 8
3 Therapeutics Development 9
Make An Enquiry @ https://www.researchmoz.us/enquiry.php?type=E&repid=1877887
3.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9
3.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14
3.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19
4 Therapeutics Assessment 23
4.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23
4.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28
4.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34
5 Companies Involved in Therapeutics Development 40
5.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 40
5.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 44
5.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 49
Continue...
About ResearchMoz
ResearchMoz is the one stop online destination to find and buy market research reports & Industry Analysis. We fulfill all your research needs spanning across industry verticals with our huge collection of market research reports. We provide our services to all sizes of organizations and across all industry verticals and markets. Our Research Coordinators have in-depth knowledge of reports as well as publishers and will assist you in making an informed decision by giving you unbiased and deep insights on which reports will satisfy your needs at the best price.
For More Information Kindly Contact:
ResearchMoz
Mr. Nachiket Ghumare,
Tel: +1-518-621-2074
USA-Canada Toll Free: 866-997-4948
Email: sales@researchmoz.us
Follow us on LinkedIn @ http://bit.ly/1TBmnVG
Follow me on @ https://marketsizeinfo.blogspot.in/
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