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ASN leads the fight to prevent, treat, and cure kidney diseases throughout the world by educating health professionals and scientists, advancing research and innovation, communicating new knowledge, and advocating for the highest quality care for patients.

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Kidney Week

Educational Symposium

Emerging Paradigms in Primary Hyperoxaluria Type 1

November 06, 2021 | 12:45 PM - 01:45 PM

Location: Simulive, Virtual Only

Session Description

Primary hyperoxaluria (PH) constitutes a group of rare inherited disorders of the liver that are characterized by the overproduction of oxalate, an end product of metabolism. High levels of oxalate are toxic because oxalate cannot be broken down by the body and it accumulates in the kidneys. Approximately 80% of all patients with PH have primary hyperoxaluria type 1 (PH1), which affects four people per million population in the United States and Europe, with an estimated 1300 to 2100 diagnosed cases. In some regions, such as the Middle East and North Africa, the genetic prevalence of PH1 is higher. Currently, the only curative treatment for PH1 is a liver transplant. In November 2020, the US Food and Drug Administration approved lumasiran, a first-in-class medication for the treatment of PH1.

Understanding the pathogenesis of inherited oxalate stone disease may lead the way to novel therapeutics and a cure. This symposium presents novel information on PH1 pathophysiology, current therapeutic options, and a way forward for the development of novel therapeutics and clinical trials.

Support is provided by an educational grant from Alnylam Pharmaceuticals, Inc.

Learning Objective(s)

  • Describe the pathophysiology of PH1
  • Discuss current and emerging PH1 treatments
  • Explain the complexities of clinical trials for patients with PH1

Learning Pathway(s)

  • Bone and Mineral Metabolism

Moderator

  • Markus J. Kemper, MD, DrMed

Presentations

  • Introduction
    12:45 PM - 12:55 PM
    Markus J. Kemper, MD, DrMed
  • The Role of Genetics and Metabolism in PH1 Pathophysiology
    12:55 PM - 01:20 PM
    Sonia Fargue, MD, PhD
  • PH1 Clinical Trials and Therapeutics
    01:20 PM - 01:45 PM
    Jaap Willem Groothoff, MD, PhD