RT @JIMD_Editors: Adding to a recent glut of #fabry papers we’ve another one, this time with a focus on ERT and there’s another #visualabst…
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In the course of Fabry disease, there is an increasing reduction in glomerular filtration rate (GFR) of up to 10–12ml/min/year. This tends to be the case even with enzyme replacement therapy. If the $SGMO gene therapy actually shows improved eGFR values, …
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RT @JIMD_Editors: Adding to a recent glut of #fabry papers we’ve another one, this time with a focus on ERT and there’s another #visualabst…
1,098 followers
A phase 3 open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease https://t.co/zZumnmG4eg Treatment burden and adherence are always an issue with ERT. A one-time gene therapy would be a leap forw…