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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.
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?
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
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.
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?
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
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