04/27/2026
PKD NEWS
The Rogosin Institute is offering a webinar this coming Thursday, April 30, 7-8:30pm, on a new potential PKD Treatment, Vertex VX-407 that is set to begin a clinical trial at Rogosin this year.
Vertex VX-407 (“the Protein Fixer"):
How it works: Think of your kidney cells like a factory that needs a specific instruction manual to produce polycystin proteins, which keep the kidneys healthy. In many people with PKD, the manual is slightly "mis-written with a typo," causing the protein to fold into the wrong shape. Because it's misfolded, the body can't use the abnormal polycystin protein. VX-407 is a small molecule that works as a protein corrector. It finds those misfolded proteins and helps them fold into the correct shape so they can get back to work, helping your kidneys.
Administration: If you qualify, this medication is taken as an oral pill.
Who Qualifies: This is a highly specialized precision drug. Currently, it is only designed for patients with a very specific subset of PKD1 gene variants. We estimate that less than 10% of the PKD population will qualify for this particular study based on their genetic profile.
Webinar Details:
Topic: VX-407 and the Genetics of PKD
Date: Thursday, April 30th
Time: 7:00 PM - 8:30 PM
Link to Join: https://nyph.zoom.us/j/98243197004 (No registration required—just click to join!)
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