Alagille Syndrome News and Research

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Alagille Syndrome is a rare inherited liver disorder seen in infants and young children. The disease is characterized by a buildup of bile in the liver due to a deficiency or absence of normal bile ducts inside the liver and a narrowing of bile ducts outside the liver. Symptoms of the disorder can include jaundice, stunted growth, fatty deposits in the skin, facial deformities, and abnormalities in the heart, eyes, vertebrae, and kidneys.
SickKids study offers new path for pediatric rare disease clinical trials

SickKids study offers new path for pediatric rare disease clinical trials

DNA origami used to unveil novel Notch receptor activation pathway

DNA origami used to unveil novel Notch receptor activation pathway

Incurable liver disease could be reversed with a single drug

Incurable liver disease could be reversed with a single drug

New source of stem cells could help treat people with Alagille syndrome

New source of stem cells could help treat people with Alagille syndrome

Deletion of an embryonic gene in liver cells completely wipes out bile ducts of newborn mice

Deletion of an embryonic gene in liver cells completely wipes out bile ducts of newborn mice

SOX9 broadly modifies the severity of Alagille syndrome liver disease in mice

SOX9 broadly modifies the severity of Alagille syndrome liver disease in mice

Scientists highlight advances and uses of organoids in gastroenterology and hepatology

Scientists highlight advances and uses of organoids in gastroenterology and hepatology

Study reveals molecular mechanisms underlying expansive arterial remodeling

Study reveals molecular mechanisms underlying expansive arterial remodeling

Scientists discover mechanism behind unusual form of tissue regeneration in the liver

Scientists discover mechanism behind unusual form of tissue regeneration in the liver

Liver disease in children with Alagille Syndrome caused by malformations of bile ducts

Liver disease in children with Alagille Syndrome caused by malformations of bile ducts

Jaw malformations in zebrafish could provide clues about hearing loss in mice and humans

Jaw malformations in zebrafish could provide clues about hearing loss in mice and humans

UConn-led researchers identify specific gene linked to Hajdu-Cheney syndrome

UConn-led researchers identify specific gene linked to Hajdu-Cheney syndrome

Researchers develop new technology to track DNA-protein binding in live cells

Researchers develop new technology to track DNA-protein binding in live cells

Phase 2 IMAGO trial of SHP625 fails to meet primary endpoints in pediatric patients with ALGS

Phase 2 IMAGO trial of SHP625 fails to meet primary endpoints in pediatric patients with ALGS

Dr. Hans Clevers receives ISSCR's McEwen Award for Innovation

Dr. Hans Clevers receives ISSCR's McEwen Award for Innovation

Lumena Pharmaceuticals initiates LUM001 clinical program in children with ALGS

Lumena Pharmaceuticals initiates LUM001 clinical program in children with ALGS

New study reveals another duet played by Notch and BMP signals

New study reveals another duet played by Notch and BMP signals

Link between Jagged-1 protein and bone formation

Link between Jagged-1 protein and bone formation

EMA COMP issues positive opinion to Albireo for A4250

EMA COMP issues positive opinion to Albireo for A4250

Embryonic signalling pathways suggest a new therapeutic approach to recover from heart attack

Embryonic signalling pathways suggest a new therapeutic approach to recover from heart attack

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