Pompe disease therapy market

Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally. There are three types of Pompe disease, which differ in severity and the ag...

Pompe disease is an inherited rare disease, often fatal with a potential to damage heart, liver and skeletal muscles. Absence of an essential protein, Due to the mutation in a gene encoding GAA, there is an absence of a critical protein leading to a complex buildup of sugar, hence, damaging heart, liver and other o...

The global Pompe disease market will experience a boost during the study period of 2019-28. Pompe disease is a rare inheritable disorder with a combined Pompe disease prevalence of 1 in 40,000, affecting males and females equally. The Pompe disease therapeutic market revolves around pharmacological treatment (wh...

An orphan disease is any disease that affects only a small percentage of the population. There can be as many as 7,000 rare diseases known to affect human life. The total number of Americans living with a rare disease is estimated at between 25-30 million. When it comes to the EU, all together, rare disease patient...

Pompe disease, an inherited rare disease, is often fatal and incapacitates the heart and skeletal muscles. Absence of an essential protein due to the mutation in a gene encoding GAA, leads to a complex buildup of sugar, hence, damaging heart, liver and other organs. The mutated gene hampers the lysosomal-mediated d...