Market Research Report
Pompe Disease: Epidemiology Forecast to 2027
|Published by||GlobalData||Product code||758948|
|Published||Content info||40 Pages
Delivery time: 1-2 business days
|Pompe Disease: Epidemiology Forecast to 2027|
|Published: November 27, 2018||Content info: 40 Pages||
Pompe disease is a rare multisystem disorder caused by pathogenic variations in the GAA gene, which contains the information for production and function of a protein called acid alpha-glucosidase (GAA). Because of the shortage of this protein (an enzyme), a complex sugar named glycogen cannot be degraded to a simple sugar like glucose. This causes the glycogen to accumulate in all kinds of tissues, but primarily in skeletal muscle, smooth muscle, and cardiac muscle, where it causes damage to tissue structure and function. Pompe disease is inherited as an autosomal recessive genetic trait.
For the 8MM, GlobalData epidemiologists provide two different forecasts for the diagnosed incident and diagnosed prevalent cases of Pompe disease-a forecast based on international registry data and a forecast adjusted for the underestimation of international registry-based data.
Based on international registry data, GlobalData epidemiologists estimated that the 8MM had 135 diagnosed incident cases of Pompe disease in 2017. GlobalData epidemiologists believe the registry underestimates the true incidence of Pompe disease. GlobalData estimates that there are 365 diagnosed incident cases of Pompe disease in the 8MM in 2017 after adjusting for the underestimation of international registry data. This is expected to increase to 373 incident cases by 2027.
GlobalData epidemiologists used the available data on Pompe disease to the best extent possible to provide the most insightful epidemiological forecast for the diagnosed incident cases and diagnosed prevalent cases of Pompe disease in the 8MM. GlobalData epidemiologists conducted an exhaustive literature review in order to find the best available data, and used the most appropriate incidence and prevalence estimates to the extent that they were available.
The Pompe Disease Epidemiology report will allow you to -