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Amicus presents positive data on Pompe drug, but could wait years for approval

  • Matthew Herper

By Matthew Herper Feb. 5, 2019

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John and Megan Crowley
Amicus CEO John Crowley and daughter Megan, who has Pompe disease. Crowley family

Amicus Therapeutics (FOLD) said Tuesday that its drug for Pompe disease appeared to benefit patients with the disease in a small trial, a modest but noteworthy milestone ahead of what will be a pivotal trial.

“This is an important next step on what’s almost a 20-year journey,” said Amicus CEO John Crowley.

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About the Author Reprints

Matthew Herper

Matthew Herper

Senior Writer, Medicine, Editorial Director of Events

Matthew Herper covers medical innovation — both its promise and its perils.

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