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Giancarlo Parenti, MD - "Pompe disease: from pathophysiology to therapy"

Associate Investigator, TIGEM and Associate Professor of Pediatrics, Department of Translational Medicine, University of Naples "Federico II", Naples, Italy
When Feb 19, 2019
from 12:00 PM to 01:30 PM
Where Tigem, Vesuvius Auditorium
Contact Name
Contact Phone 081-19230659
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Abstract
Over the past decade multiple therapeutic approaches have been developed to treat Pompe disease, a lysosomal storage disorder due to the deficiency of acid alpha-glucosidase. Research in this field has been mainly focused on the development of efficacious therapeutic agents, all based on increasing the activity of the missing enzyme, such as enzyme replacement therapy, pharmacological chaperone therapy, and gene therapy. The improved knowledge on the pathophysiology of the disease and on the secondary impairments of cellular pathways elicited by glycogen storage, suggests that these events may represent additional and novel targets for therapy. Correction of secondary abnormalities of the autophagic pathway and of response to oxidative stress, may contribute to a better efficacy of existing therapies.

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