PKD Foundation
@pkdfoundation.bsky.social
Dedicated to finding treatments and a cure for polycystic kidney disease (PKD). www.pkdcure.org
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.
November 3, 2025 at 3:38 PM
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...
Read more about this exciting development: www.nature.com/articles/s41...
PKD1 5’UTR variants are a rare cause of disease in ADPKD and suggest a new focus for therapeutic development - European Journal of Human Genetics
European Journal of Human Genetics - PKD1 5’UTR variants are a rare cause of disease in ADPKD and suggest a new focus for therapeutic development
www.nature.com
October 16, 2025 at 2:39 PM
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...
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.
October 16, 2025 at 2:39 PM
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.
October 16, 2025 at 2:39 PM
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.
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.
September 29, 2025 at 6:00 PM
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.
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.
September 29, 2025 at 6:00 PM
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.
September 22, 2025 at 2:49 PM
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/
Advocacy
Advocacy - With PKD Connect, no one will ever face polycystic kidney disease alone. Because patients, family and loved ones will always be connected to others who understand firsthand what you’re goin...
pkdcure.org
September 17, 2025 at 2:03 PM
Join our Congressional Champions in their advocacy efforts by becoming part of our Advocacy Champions Network! Learn more at pkdcure.org/advocacy/
*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.
September 9, 2025 at 1:35 PM
*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.
*Our 4-part educational series is designed to help patients and families better understand the latest KDIGO recommendations for ADPKD management.
September 9, 2025 at 1:35 PM
*Our 4-part educational series is designed to help patients and families better understand the latest KDIGO recommendations for ADPKD management.
*The Kidney Disease: Improving Global Outcomes (KDIGO) 2024 Guidelines for ADPKD provide the latest evidence-based recommendations to help patients, caregivers, and healthcare providers navigate the complexities of autosomal dominant polycystic kidney disease (ADPKD).
September 9, 2025 at 1:35 PM
*The Kidney Disease: Improving Global Outcomes (KDIGO) 2024 Guidelines for ADPKD provide the latest evidence-based recommendations to help patients, caregivers, and healthcare providers navigate the complexities of autosomal dominant polycystic kidney disease (ADPKD).