Rare Disease Report: The FDA Decision for Pompe Disease Therapy
October 28, 202213m 25s
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Show Notes
The FDA is expected to announce the final decision for the dual therapy AT-GAA, which uses miglustat in combination with cipaglucosidase alfa for treating Pompe disease, an inherited and often times fatal disorder. Expert Barry Byrne discusses the characteristics of the condition and what this FDA decision will mean for treatment.