LONDON--(BUSINESS WIRE)--Technavio has announced their latest pipeline analysis report on the Gaucher disease market. The report comprises an in-depth analysis of the pipeline molecules under investigation within the defined data collection period to treat Gaucher disease.
This report 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 in the coming years.
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Gaucher disease - market overview
Gaucher disease is an autosomal recessive inherited disorder of metabolism where a type of fat (lipid) called glucocerebroside cannot be adequately degraded. The common symptoms of the disease include an enlarged liver or spleen, low levels of circulating red blood cells, clotting blood cells and skeletal abnormalities. In rare cases, the disease also affects the nervous system and nerve cells. The disease is classified into type 1, type 2 and type 3. Of these, type 1 is the most common and this does not affect the nervous system.
According to a senior analyst at Technavio for infectious and rare diseases research, “The disease is caused by a recessive mutation in the GBA gene located on chromosome 1 and affects both males and females. About one in 100 people in the United States are carriers of the most common type of Gaucher disease.”
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Gaucher disease – market segmentation
This market research report segments the Gaucher disease market based on therapies employed (monotherapy and combination therapy), mechanism of action (enzyme replacement, glucosylceramide synthase inhibitors, histone deacetylase, molecular chaperone modulators), therapeutic modalities (small molecule and recombinant enzyme), and RoA (intravenous and oral).
The IV RoA is expected to account for over 41% of the market share. IV delivers liquid substances directly into the vein. Based on therapeutic modality, recombinant enzyme accounted for 47% of all molecules in the current pipeline. With respect to MoA, over 47% of the molecules in the pipeline work towards the replacement of glucocerebroside as the disease occurs due to deficiency of this enzyme.
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