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Xtalks, Online
2025-12-04
This webinar will explore polycystic kidney disease (PKD) with a focus on autosomal dominant PKD (ADPKD), including clinical presentations, considerations for conducting clinical trials in this population and the future of ADPKD treatment.
In the United States, approximately 600,000 people have PKD1, making it the fourth leading cause of kidney failure, and there is no cure presently available.2 Men and women are equally at risk for this inherited disorder, and about 90% of PKD cases are ADPKD, meaning only one copy of the abnormal gene, either PKD1 or PKD2, is needed to cause the disease. Globally, ADPKD affects approximately 12 million people3 and is the most common cause of genetic kidney failure. Autosomal recessive PKD, or ARPKD, means that both parents must have and pass down the PKHD1 mutation to their child; ARPKD is extremely rare and only occurs in about one in 25,000 people.
Since 2014, genetic testing for ADPKD has improved in accuracy, availability and affordability, leading to higher usage; additional genes outside of PKD1 and PKD2 have also been associated with this disorder, and significant interest in improved outcomes for patients persists. There has been a noticeable increase in clinical trial activity related to potential new therapies for ADPKD over the last several years; tolvaptan was approved in 2015 and 2018 by Health Canada, the European Medicines Agency (EMA) and the US Food and Drug Administration, respectively, and there are more drugs in development for ADPKD now than ever before.4
The recently released Kidney Disease: Improving Global Outcome guidelines for ADPKD highlight the need for improved biomarkers of ADPKD progression and have led to focused research in the development of serum proteomics-based outcome prediction models.5 Beyond proteomics, another growing body of research surrounds ciliopathies, or primary cilia dysfunction. As ciliopathies lead to a wide range of disorders, the most common of which is ADPKD, and primary cilia are critical for the regulation of cellular metabolism, glutamine utilization via asparagine synthetase (ASNS) is another potential area of interest as a target for therapy.
The featured speakers will highlight the current landscape for ADPKD, factors to consider for clinical trials and what the future may hold for possible new therapies. They will consider the novel challenges and opportunities that these evolving questions present in addressing the urgent need for increasing the number of safe and effective treatments for people living with ADPKD.
Register for this webinar to learn how polycystic kidney disease clinical trials are shaping the future of ADPKD treatment.
Keywords: Clinical Development, Clinical Research, Clinical Trials, CRO, Drug Development, Kidney Disease, Kidney Disease Research, Kidney Failure, Renal, Renal Disease, Therapeutic Areas, Therapeutics
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Organized by:
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Xtalks |
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Invited Speakers:
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Dr. Sayna Norouzi, Associate Professor of Medicine, Founder & Director of both the Polycystic Kidney Disease Clinic and the Glomerular Disease Clinic at Loma Linda University Medical Center
Dr. Hannah Troutman, Nephrologist and Associate Medical Director at the PPD clinical research business of Thermo Fisher Scientific
(Moderator) Sarah Dixon Stump, MS, Project Oversight Senior Director at the PPD clinical research business of Thermo Fisher Scientific
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Deadline for Abstracts:
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2025-12-04
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Registration:
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Free Registration
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E-mail:
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tristan@xtalks.com
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