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Expert Interview
Slingshot members are talking to an expert! The topic is:
A review of AT-GAA from Amicus Therapeutics in late-onset Pompe Disease ahead of the PDUFA
Ticker(s): FOLDWho's the expert?
Institution: Northwestern
- Professor of Pediatrics (Genetics, Birth Defects, and Metabolism) at Northwestern University Feinberg School of Medicine & Director, PKU Program.
- Currently manages 40 patients with Pompe disease and is familiar with the literature on alglucosidase alfa (AT2221). Also, manages 320 patients with Phenylketonuria (PKU).
- Participated in numerous clinical trials of new therapies for metabolic disorders including PKU, lysosomal storage disorders and fatty acid oxidation defects and has authored over 200 articles in the peer-reviewed literature.
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