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Revolution in Diabetes Treatment: Repurposed Drug Shows Promise

Recent research led by Indiana University School of Medicine in collaboration with the University of Chicago Medicine presents exciting future possibilities for the management of type 1 diabetes and the potential reduction of insulin dependency. The researchersโ€™ findings, published in Cell Reports M
Raghavendrag Mirmira Fangqian Ouyang Lindaa Dimeglio Abhishek Kulkarni Carmella Evans Molina Lori Blanchfield

Novartis Pharma AG: Novartis Phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients

Data at EBMT show primary endpoint met - estimated* 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2 g/dL or more hemoglobin-level increase from baseline
South Korea France General United States Satoshi Sugimoto Julie Masow Giuseppe Moscati

Novartis Pharma AG: Novartis presents pivotal Phase III APPLY-PNH data at ASH demonstrating investigational oral monotherapy iptacopan superiority over anti-C5

Trial met both primary and most secondary endpoints, showing iptacopan provided transfusion-free hemoglobin-level increases in vast majority of adult paroxysmal nocturnal hemoglobinuria (PNH) patients
France General United States Richard Jarvis Isabella Zinck Michael Meo Satoshi Sugimoto

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Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G

Posted December 16th, 2020 for Novartis Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder, resulting in debilitating symptoms that can impact patientsโ€™ quality of life 1โ€“3 C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need 4-5 With potential to be the first oral treatment for a range of complement-driven diseases, complement factor B inhibitor iptacopan targets the underlying...
United States Eric Althoff Isabella Zinck Samir Shah Thomas Hungerbuehler Phil Mcnamara

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