DUBLIN--(BUSINESS WIRE)--The "Mucopolysaccharidosis
I (Hurler Syndrome) Pipeline Analysis 2018 (H1) - Focusing on Clinical
Trials and Results, Drug Profiling, Patents, Collaborations, and Other
Recent Developments" report has been added to ResearchAndMarkets.com's
Mucopolysaccharidosis I (Hurler Syndrome) Pipeline Analysis 2018 - Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Developments.
Mucopolysaccharidosis I (Hurler Syndrome) Pipeline Analysis report covers 11 drugs currently in different phases of development. Hurler Syndrome is an inherited lysosomal storage disease caused by defect in IDUA gene. The defect in the gene results in deficiency of alpha-L iduronidase enzyme which helps in helps break down long chains of sugar molecules called glycosaminoglycans. Without the enzyme glycosaminoglycans build up and damage of tissues and organs. The disease is characterised by skeletal abnormalities, cognitive impairment, heart disease, respiratory problems, enlarged liver and spleen, characteristic facies and reduced life expectancy.
The report provides Mucopolysaccharidosis I (Hurler Syndrome) treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region.
Major industry players profiled as part of the report are ArmaGen, Angiochem and JCR Pharmaceuticals Co Ltd among others.
Key Topics Covered:
2. Disease Overview
3. Executive Summary
4. Market Dynamics
5. Pipeline Analysis/Outlook
6. Company Profiling
- Sangamo Therapeutics, Inc.
- Eloxx Pharmaceuticals
- REGENXBIO Inc.
- Crispr Therapeutics
- Jupiter Orphan Therapeutics, Inc.
- Orchard Therapeutics
- ProQR Therapeutics
- JCR Pharmaceuticals Co Ltd
For more information about this report visit https://www.researchandmarkets.com/research/prvrw5/mucopolysaccharido?w=4