LONDON--(BUSINESS WIRE)--Technavio has announced its latest pipeline analysis report on the Pompe disease market. The report comprises an in-depth analysis of the pipeline molecules under investigation within the defined data collection period to treat Pompe 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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Pompe disease - market overview
Pompe disease market is also known as type II glycogen storage disease or acid maltase deficiency disease. It is an autosomal recessive disorder caused by an accumulation of glycogen skeletal and cardiac muscle due to the deficiency of the lysosomal acid alpha-glucosidase enzyme. The glycogen accumulation impairs the ability of muscles to function normally. Pompe disease can also be life-threatening if breathing and heart muscles get affected.
According to a senior analyst at Technavio for infectious and rare diseases research, “Pompe disease occurs due to GAA gene mutations. GAA gene is located on the 17th chromosome and it delivers instructions for alpha-glucosidase enzyme production. The age of disease onset and disease severity are related to the degree of alpha-glucosidase deficiency.”
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Pompe disease – market segmentation
This market research report segments the Pompe disease market based on therapies employed (monotherapy, combination therapy, and unknown), therapeutic modalities (gene therapy, small molecule, and recombinant enzyme), and RoA (IV, oral, IV + oral, and unspecified).
The IV RoA is expected to account for around 63% of the market share. IV delivers liquid substances directly into the vein. In the current pipeline, ten molecules are for IV RoA. Base on therapies employed, monotherapy which involves the use of a single drug for treatment is expected to account for 69% of the market. In the current pipeline, 11 molecules are for monotherapy.
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