STAT+: In a medical first, Vertex drug successfully targets the underlying cause of a genetic kidney disease
An experimental pill from Vertex Pharmaceuticals reduced levels of a damaging urine protein by nearly half in a small clinical trial — encouraging evidence that a novel medicine can arrest the underlying genetic cause of a chronic kidney disease that progresses rapidly to kidney failure.
Vertex called the mid-stage study results “unprecedented” and said it was working quickly to advance the drug, called VX-147, into a pivotal clinical trial, pending discussions with regulators.
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