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When two of John Crowley’s children were diagnosed with the rare and debilitating Pompe disease, he founded a company that helped develop a successful treatment. Now, he’s running another biotech — and clinical trial data presented Wednesday suggest that it’s on the way to developing a more effective drug for the same disease.

The new drug from Amicus Therapeutics (FOLD) appears to deliver greater improvements in muscle and lung function for people with Pompe, according to interim data from a small, mid-stage study.


“With these data, we have increasing confidence that this is a differentiated approach to treating Pompe. We are making people stronger,” said Crowley, Amicus’ CEO, in an interview with STAT ahead of the study presentation at the World Muscle Society meeting underway in France.

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