Cystic Fibrosis Foundation’s Post

The FDA has officially approved Xolremdi (mavorixafor) as a treatment for WHIM syndrome, a rare genetic immune disorder characterized by warts, hypogammaglobulinemia, infections, and myelokathexis. WHIM syndrome severely impacts patients’ quality of life by leaving them prone to infections and immune system dysfunction. Xolremdi is a groundbreaking medication, designed to increase the count of mature neutrophils and lymphocytes in the blood. This targeted approach is a game-changer, especially for patients aged 12 years and older, as it addresses the root cause of their immune deficiency. This approval is a significant step forward in personalized medicine, offering new hope to individuals with limited treatment options. It’s always inspiring to witness advancements in rare disease therapies, highlighting the dedication of researchers and healthcare professionals in making a real difference. For more details, visit the FDA’s official page on this approval: https://lnkd.in/daDkP4ZM

Phase 2b Study of Ianalumab in Sjögren's A 52-week has demonstrated the safety and efficacy of ianalumab, a dual B cell depleter) in patients with active Sjögren's disease (SjD).

Phase 2b Study of Ianalumab in Sjögren's A 52-week has demonstrated the safety and efficacy of ianalumab, a dual B cell depleter) in patients with active Sjögren's disease (SjD).

For decades, steroids have been considered a necessary evil to control ANCA-associated vasculitis. Yet today, more patients die from complications associated with glucocorticoid treatment than the underlying disease. Get a summary of the most comprehensive picture to date of evolving diagnostic and therapeutic strategies for this rare but devastating #AutoimmuneDisorder: https://hubs.li/Q02rhSdw0 #Vasculitis #AutoimmuneDiseaseManagement #SteritasGTI #SteroidToxicity #SteroidToxicityAwareness #VasculitisTreatment #SteroidInducedComplications

https://lnkd.in/eqpP_DPX From 2013…once you understand inflammation’s role in vascular disease and GLP-1s role in impacting inflammation, the potential benefits become clearer. The next step is to see if there is benefit in patients with established AD.

For decades, steroids have been considered a necessary evil to control ANCA-associated vasculitis. Yet today, more patients die from complications associated with glucocorticoid treatment than the underlying disease. Get a summary of the most comprehensive picture to date of evolving diagnostic and therapeutic strategies for this rare but devastating #AutoimmuneDisorder: https://hubs.li/Q02qxYW20 #Vasculitis #AutoimmuneDiseaseManagement #SteritasGTI #SteroidToxicity #SteroidToxicityAwareness #VasculitisTreatment #SteroidInducedComplications

For patients with metabolic dysfunction-associated steatotic liver disease (MASLD) and diabetes, glucagon-like peptide 1 receptor agonist (GLP-1 RA) use is associated with a reduced risk for cirrhosis and for cirrhosis complications and mortality. https://brnw.ch/21wN3R5

🚀 Continuing Your Pharma Education: Intestinal Diseases – From Inflammatory Conditions to Intestinal Cancer 🩺 In our latest article, we explore intestinal diseases like IBS, IBD, and intestinal cancer, discussing causes, symptoms, and modern treatment approaches. Learn about the latest pharmaceutical strategies for managing these complex conditions. #IntestinalDiseases #IBD #CeliacDisease #PharmaEducation #IntestinalCancer #GutHealth #NewToPharma #PharmaceuticalIndustry #Gastroenterology #MedicalRepresentative #PharmaColleagues #Health #MedicalStudent #HealthTips Read the full article now and stay tuned for more! 👇 https://lnkd.in/dD4f2cEw

https://lnkd.in/eyGQkEBX Semaglutide for liver disease? We now have a drug that impacts liver, heart and kidney disease. We also know that the Fib-4 score can suggest underlying disease in these three illnesses. Perhaps it’s time to really explore how INFLAMMATION and FIBROSIS is the common denominator in common chronic diseases and use targeted therapies against these two. Follow the inflammasome.

Safety Review Committee (SRC) approves single ascending dose 4mg cohort in Phase 1 trial of PMC-403, preparing for multiple ascending dose 4 mg cohort. PMC-403 is being explored in broader therapeutic areas, including rare vascular disease, systemic capillary leak syndrome (SCLS) and kidney diseases. https://lnkd.in/g3Qiexr5