『S2 Ep8: The FDA Decision for Pompe Disease Therapy』のカバーアート

S2 Ep8: The FDA Decision for Pompe Disease Therapy

S2 Ep8: The FDA Decision for Pompe Disease Therapy

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2026年5月12日まで。4か月目以降は月額1,500円で自動更新します。

概要

 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.
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