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Subject: SVY

2018 Mucopolysaccharidosis Disorders Drug Development Pipeline Review


DUBLIN, Aug. 17, 2018 /PRNewswire/ --

The "Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018" report has been added to ResearchAndMarkets.com's offering.

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This report "Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018" 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

Reasons to buy

Key Topics Covered:

1 Tables & Figures

2 Introduction

3 Therapeutics Development

4 Therapeutics Assessment

5 Companies Involved in Therapeutics Development

6 Dormant Projects

7 Discontinued Products

8 Product Development Milestones

9 Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/research/d5qwt7/2018?w=5

Media Contact:

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SOURCE Research and Markets


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