Hurler Syndrome | A Pipeline Analysis Report 2018 | Technavio
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Technavio has published a new report on the drug development pipeline for hurler syndrome, including a detailed study of the pipeline molecules. (Graphic: Business Wire)
This report by Technavio presents a detailed analysis of the market, including regulatory framework, drug development strategies, recruitment strategies, and key companies that are expected to play an essential role in the growth of the market.
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Hurler syndrome: Market overview
Hurler syndrome is an autosomal recessive disorder, which is caused by the mutation in the iduronidase (IDUA) gene. The two common types of mutation observed in the IDUA gene are W402X and Q70X that consist of mutant alleles. Hurler syndrome is also called as mucopolysaccharidosis type I (MPSI), which happens when the body lacks the alpha-L-iduronidase enzyme, which is needed to break down long chains of sugar molecules.
According to a senior analyst at Technavio for research on infectious and rare diseases, “The body of people suffering from Hurler syndrome fails to produce an enzyme called lysosomal alpha-L-iduronidase. In the absence of this enzyme, GAGs get accumulated significantly, which will damage the organs, including the heart. Many companies are concentrating on developing molecules for the treatment of neurological complications such as Hurler syndromes.”
Hurler syndrome: Segmentation analysis
This market research report segments the Hurler syndrome market based on therapies employed (monotherapy and unknown), RoA (oral, intravenous, intraperitoneal, parenteral, subcutaneous, and unknown), therapeutic modality (gene therapy, small molecule, RNA, stem cell, cell therapy, enzyme, monoclonal antibody, and peptide), targets (hepatic cells, iduronidase gene and sirtuins, insulin receptor, and unknown), MoA (iduronidase replacement, enzyme replacement, cell replacement, and iduronidase gene modulator and sirtuins activator), and recruitment status (recruiting, active not recruiting, completed, and NA).
Monotherapy includes the use of a single drug to treat a disorder or a disease. In the current pipeline, around 63% of the molecules that are being investigated for the treatment of Hurler syndrome are monotherapy.
In case of intravenous RoA, the drug substance is delivered directly into the vein. About 44% of the total molecules that are in the current pipeline are expected to be administered intravenously.
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Some of the key topics covered in the report include:
Scope of the Report
Drug Development Landscape
- Drugs under development
- Indications coverage
Drug Development Strategies
- Therapies employed
- Therapeutic modality
- Geographical coverage
- Recruitment status
- Recruitment volume
- Type of players
- Company Overview
Discontinued or Dormant Molecules
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Source: Technavio Research