Nephrologists

China’s NMPA Accepts ALGS New Drug Application for Maralixibat

China’s National Medical Products Administration (NMPA) accepted the New Drug Application (NDA) for CAN108 (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 1 year and above. “That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for [ALGS], our first candidate in…

Phase 3 Trial Testing Odevixibat for Alagille Syndrome Is Underway

Albireo Pharma is continuing to enroll participants for its phase 3 clinical trial called ASSERT to test the safety and efficacy of their nonsystemic ileal bile acid transport inhibitor odevixibat (Bylvay™) in patients with Alagille syndrome. The trial remains on track for topline results by the end of 2022. “Bylvay launch uptake has been faster…

Renovascular Hypertension Might Be a Presenting Symptom in ALGS

Researchers presented a rare case of an individual complaining of renovascular hypertension who was diagnosed with Alagille syndrome (ALGS), as published in the Journal of Genetic Medicine. “In contrast to most ALGS cases where the symptoms at presentation are related to congenital liver disease and heart anomalies within the first year of life, the clinical…

New Insights on Therapeutic Emergencies in ALGS

Researchers addressed scenarios that covered diagnostic and therapeutic emergencies in children with liver diseases such as Alagille syndrome (ALGS), as published in the Journal of Hepatology. These could help the early diagnosis and timely management of pediatric liver conditions. Infants may be affected by a wide range of liver disease-causing conditions. In some instances, jaundice…

Skin Changes Can Aid in Early Diagnosis of Alagille Syndrome

Doctors detailed the skin changes that occur in cases of cirrhosis, as published in the Journal of Clinical and Experimental Hepatology. Cirrhosis or liver fibrosis caused by long-term liver damage can be caused by a number of conditions including viral infections, metabolic disorders, and biliary tract diseases such as Alagille syndrome. Alagille syndrome is caused…

Could Organoid Cultures Help Better Understand Alagille Syndrome?

Researchers reported the development of organoid cultures initiated from bile samples obtained during routine clinical procedures and showed that these organoids are able to repopulate human extrahepatic bile duct scaffolds, as published in Clinical and Translational Medicine. Alagille syndrome is a disease characterized by intrahepatic bile duct paucity. In the present study, a team of…

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