With your support, we can create a future with fewer missed milestones and more meaningful memories for families affected by PKD.

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This briefing is an important step toward restoring these critical investments.

Join us by contacting your lawmakers today to advocate for PKD research! pkdcure.org/RestoreCDMRP

The CDMRP is a Department of Defense program that funds research on diseases to drive new treatments and cures. In March, the program’s funding was cut by 57%. The CDMRP is an essential source of funding to find treatments and a cure for PKD, having provided $80 million for research since 2020.

In 2024, the first procedures lasted only one to two months. It’s a big step forward and a sign of hope for the 90,000 people in the U.S. waiting for a kidney transplant, including people with PKD.

Modifying uORFs to increase polycystin production could open the door for a promising new class of potential therapies for ADPKD.

Read more about this exciting development: www.nature.com/articles/s41...

Researchers in Australia and the UK have found that targeted changes to upstream open reading frames (uORFs) in the genetic region just before the PKD1 gene can also increase or decrease polycystin levels.

Polycystins are proteins essential to healthy kidney function. In patients with ADPKD, mutations of the PKD1 or PKD2 gene reduce polycystin production by half, ultimately leading to cyst formation.

With VX-407, it functions much like other small-molecule drugs Vertex has already developed and had approved for cystic fibrosis.

This proof-of-concept study will include 24 participants from around the world, each taking VX-407 for 52 weeks. To qualify, patients must undergo genetic testing.

VX-407 is designed to target the source of ADPKD in patients with changes in the PKD1 gene. The way this investigational study drug works in the body is different from other treatments available for ADPKD.

This webinar is supported by a sponsorship from Otsuka. The PKD Foundation retains full control over the content and speaker selection to ensure unbiased, high-quality education for our community.

Join our Congressional Champions in their advocacy efforts by becoming part of our Advocacy Champions Network! Learn more at pkdcure.org/advocacy/