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(Last time: the missing enzyme in Fabry is alpha-galactosidase A.)
When that enzyme goes missing, one particular molecule builds up in the blood and becomes the fingerprint doctors track to measure the disease. Cut it roughly in half, and you've shown a therapy is working.
What's this telltale biomarker? Drop your guess in the comments below.
Follow the whole series: https://b2f.bz/hzlEB
@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung
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