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Pompe disease is an extremely rare disorder in Japan, making the task of assessing epidemiology, diagnosis, and understanding patient needs complex.
This white paper, researched by Informa Pharma Intelligence highlights important insights that will support the development of new treatment options.
Clinical characteristics of Pompe disease
Diagnostic testing processes
Patient journey from symptom onset to diagnosis
Diagnostic challenges associated with Pompe disease
Currently available drug therapy and unmet needs
Download this white paper for the latest research into the current state of this rare disease.
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