Partial correction of cystinuria type A in mice via kidney-targeted transposon delivery We used kidney-targeted, non-viral, transposon-mediated gene delivery to express the mouse Slc3a1 transgene in one kidney of cystinuria type A (Slc3a1−/−) mice. We found a 44% reduction in urinary cystine concentration at 154 days post-gene transfer, although there was no significant effect on cystine stone [...]
Non-viral gene therapy to treat cystinuria Aug. 4, 2025 Original Story (Unfortunately the story is buried behind a paywall so we may need to wait for the study to be published before we can see more.) In an effort to develop next-generation treatments for cystinuria, researchers at Vanderbilt University and Tennessee Valley Health Services have [...]
Cystinuria Pipeline Insight 2025: Redefining Treatment through Precision Therapies and Rare Disease Innovation | DelveInsight Original Story 07-21-2025 04:20 PM CET | Health & Medicine Press release from: DelveInsight DelveInsight's "Cystinuria - Pipeline Insight, 2025" provides a detailed examination of the ongoing therapeutic R&D landscape targeting cystinuria, a rare inherited disorder characterized by defective renal reabsorption of [...]
Cystinuria Awareness Day in Dixie! Holly C Hey! My name is Holly, and I am 41 years old, and am from Hanceville, AL. , about an hour north of Birmingham. I was diagnosed with Cystinuria at the age of 14, so it will be 27 years this fall since I was diagnosed. One [...]
My story and I’m still alive to tell it Brenda I had my first kidney stone when I was 17. I was at a slumber party, passed out, and woke up the next morning in terrible pain. They cut through my left side, from my belly to my back — and that was [...]
Cystinuria Pipeline Insight 2025: Redefining Treatment through Precision Therapies and Rare Disease Innovation | DelveInsight Original Story Professor Richard Coward from the University of Bristol has received a Kidney Research UK research project grant of £230,000 to look at developing drugs that target the transportation pathway of cystine in kidney cells involved in cystinuria. The problem with cystinuria causing kidney stones Cystinuria is a rare inherited disease which causes [...]
“Go big or go small”: Current and emerging tools in kidney stone management Original Story Key Takeaways Recent advancements in kidney stone management include novel access sheaths and miniaturized scopes, enhancing treatment options for patients. AI integration in kidney stone management offers potential for improved imaging standardization and enhanced patient education. SHOW MORE “The technology [...]
Sexual Dimorphism in Cystinuria- The Mitochondria Link Original Story Unravelling the role of mitochondrial Slc3a1 in regulating mitochondrial functions and sexual dimorphism in cystinuria CHINA, March 28, 2025 /EINPresswire.com/ -- Cystinuria is the most common inheritable cause of kidney stone disease, characterized by impaired reabsorption of cystine and dibasic amino acids in the renal proximal tubules. It [...]
New US product launch, VENXXIVA™ (tiopronin) Delayed-Release Tablets for the treatment of cystinuria Original Story BOSTON--(BUSINESS WIRE)--Cycle Pharmaceuticals Limited (Cycle) announced today the launch of VENXXIVA (tiopronin) Delayed-Release Tablets, for the treatment of cystinuria, approved by the US Food and Drug Administration (FDA).1 VENXXIVA is launched in partnership between Cycle and Torrent Pharma Inc. (Torrent), and [...]
Advicenne Reaches a Significant Milestone With the US FDA in the Development of ADV7103 in Cystinuria Original Story PARIS--(BUSINESS WIRE)--Regulatory News: Advicenne (Euronext Growth Paris ALDVI – FR0013296746) a specialty pharmaceutical company dedicated to the development and commercialization of innovative treatments for those suffering from rare renal diseases, provides an update on recent exchanges with [...]
