Barmherzige Brüder

NEW YORK, May 17, 2024 /PRNewswire/ -- Renal Research Institute (RRI), a global leader in dialysis and nephrology research and a subsidiary of Fresenius Medical Care AG, today announced together with the Karolinska Institutet a joint symposium, "Prevention of CKD progression: the impact of new therapeutics." The Karolinska Institutet is one of the world's leading medical universities. Medical experts from across the globe will discuss clinical trials, prescription practices, pharmaco-epidemiology and more at this workshop in Stockholm on May 22. With chronic kidney disease (CKD) affecting about 1 in 10 adults worldwide, new therapeutics have been developed to potentially delay its progression. Notable among these are glucagon-like peptide-1 receptor agonists (GLP-1 RA) and sodium-glucose cotransporter-2 inhibitors (SGLT2i). "In the realm of chronic kidney disease management, GLP-1 RAs and SGLT2is have shown promising results in clinical trials," said Dr. Peter Kotanko, Head of Biomedical and Evidence Generation and Research Director, Renal Research Institute. "Our symposium aims to further advance the conversation on the potential of these new therapeutics, bringing together internationally recognized experts in diabetes, obesity, kidney and cardiovascular medicine to pave the way for improving patient care." The symposium aims to explore these medications and their potential impact on CKD progression in the light of the clinical trials, bringing together global medical experts and fostering an open dialogue to advance knowledge on the research through Q&A sessions, while simultaneously providing a comprehensive overview of the current landscape and future developments in the field through specialized presentations from experts. To learn more about RRI educational material and events, including past symposiums, visit the RRI website at . About Peter Kotanko, MD, FASN Head of Biomedical Evidence Generation and Research Director, Renal Research Institute Researcher and scholar Dr. Peter Kotanko leads RRI's scientists and fellows, heading the Institute's research initiatives to improve patient outcomes and quality of life. His prolific research career comprises more than 400 research papers, book chapters, and scholarly works. With over 20 years of clinical background and expertise in physiology, internal medicine, and nephrology, Dr. Kotanko is an adjunct professor of medicine and nephrology at the Icahn School of Medicine at Mount Sinai, New York. He is former vice chair of the Department of Internal Medicine, Barmherzige Brüder Hospital in Graz, Austria. About Renal Research Institute (RRI) Renal Research Institute (RRI), a subsidiary of Fresenius Medical Care, achieves its mission to improve the outcomes of patients with kidney disease through research and innovation. RRI's highly developed and specialized expertise in computational biomedicine, translational clinical and biomedical research, and data analytics underscore the Institute's track record of thinking outside the box and identifying high-value areas. Through alliance and collaboration agreements, RRI has forged strategic research relationships with leading universities in the Americas, Asia, and Europe, fostering RRI's position at the forefront of research activities in the field of dialysis and nephrology. For more information, visit the Renal Research Institute's website at . About Fresenius Medical Care: Fresenius Medical Care is the world's leading provider of products and services for individuals with renal diseases of which around 4.1 million patients worldwide regularly undergo dialysis treatment. Through its network of 3,862 dialysis clinics, Fresenius Medical Care provides dialysis treatments for approx. 325,000 patients around the globe. Fresenius Medical Care is also the leading provider of dialysis products such as dialysis machines or dialyzers. Fresenius Medical Care is listed on the Frankfurt Stock Exchange (FME) and on the New York Stock Exchange (FMS). Media Contact Kirsten Stratton T +1 781 929 8096 [email protected] SOURCE Renal Research Institute

BERLIN & COPENHAGEN--(BUSINESS WIRE)-- MyoPax has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its regenerative cell therapy in Exstrophy-Epispadias Complex (EEC). MyoPax aims to repair the congenital muscle defect associated with EEC using its cutting-edge highly regenerative muscle stem cell product. EEC is a severe newborn condition within the uro-rectal congenital malformation spectrum impacting continence, sexual and renal function. MyoPax's cell therapy uses patient-specific muscle stem cells, harnessing their regenerative potential through a proprietary and patented stem cell technology to repair the patient’s urinary sphincter muscle defect. The first-in-human MuST trial is being conducted in Germany with sponsorship from the Charité Universitätsmedizin Berlin under the guidance of Prof. Simone Spuler. Prof. Wolfgang Rösch, renowned expert and pediatric urologist, will lead one of the study centers at the Hospital Barmherzige Brüder St. Hedwig in cooperation with the University of Regensburg: "This novel approach has the potential to profoundly change the treatment of Exstrophy-Epispadias Complex. We look forward to participating in the upcoming clinical trial and working together towards the goal of improving the lives of these young patients and their families." The FDA's Orphan Drug Designation represents a significant milestone, reaffirming the company's commitment to advancing innovative solutions for rare diseases. This designation provides MyoPax with several benefits, including market exclusivity and assistance with clinical trial protocols. "We are delighted to receive FDA Orphan Drug Designation for our regenerative cell therapy for Exstrophy-Epispadias Complex," stated Dr. Verena Schöwel-Wolf, CEO of MyoPax. "This recognition validates the potential of our groundbreaking technology to address unmet medical needs and improve the lives of patients living with muscle disorders." About the MuST trial, registered under NCT04729582 EEC is a rare, complex congenital malformation of the genital and urinary organs. A defined sphincter muscle defect causes urinary incontinence and can be only incompletely corrected by current treatment, which requires multiple reconstructive surgeries and lifelong management. The first-in-human trial with the lead candidate (Satori-01) targets this orphan disease and current unmet need. The phase 1/2a trial is aimed at repairing the EEC sphincter defect and will investigate both safety and efficacy. The trial is sponsored by the Charité Universitätsmedizin and financed by the German Federal Ministry of Education and Research and the ForTra GmbH of the Else-Kröner-Fresenius Foundation. About Orphan Drug Designation: The Orphan Drug Act defines rare diseases as those affecting fewer than 200,000 individuals in the United States. Enacted by Congress in 1983, this legislation aims to encourage the development of medications for treating such diseases. The Orphan Drug Designation program grants orphan status to drugs and biologics intended for rare diseases that satisfy specific requirements. This designation brings along various incentives, which include tax credits for eligible clinical trials, exemption from user fees, and potential for a seven-year period of market exclusivity following approval. About MyoPax: MyoPax is a biotech start-up recently founded in Berlin and Copenhagen with a focus on innovative regenerative therapies for debilitating muscle disorders. It was spun out of the Charité Universitätsmedizin Berlin and the Max Delbrück Center in the Helmholtz Association and joined the BioInnovation Institute in Copenhagen with pre-seed funding. Leveraging its proprietary muscle stem cell technology and cutting-edge gene editing technologies, MyoPax aims to transform the treatment landscape and the quality of life for patients affected by muscle disorders.