Alagille Syndrome News and Research RSS Feed - Alagille Syndrome News and Research

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.
Lumena Pharmaceuticals initiates LUM001 clinical program in children with ALGS

Lumena Pharmaceuticals initiates LUM001 clinical program in children with ALGS

Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome. The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S. [More]
New study reveals another duet played by Notch and BMP signals

New study reveals another duet played by Notch and BMP signals

A small ensemble of musicians can produce an infinite number of melodies, harmonies and rhythms. So too, do a handful of workhorse signaling pathways that interact to construct multiple structures that comprise the vertebrate body. [More]
Link between Jagged-1 protein and bone formation

Link between Jagged-1 protein and bone formation

Researchers from the University of Pennsylvania School of Veterinary Medicine have discovered that a protein called Jagged-1 stimulates human stem cells to differentiate into bone-producing cells. [More]
EMA COMP issues positive opinion to Albireo for A4250

EMA COMP issues positive opinion to Albireo for A4250

Albireo AB, a biopharmaceutical company specializing in gastroenterology, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for the company's lead hepatology candidate, A4250. [More]
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

Almost a century after it was discovered in fruit flies with notches in their wings, the Notch signalling pathway may come to play an important role in the recovery from heart attacks. In a study published today in Circulation Research, scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, are the first to prove that this signalling pathway targets heart muscle cells and thus reveal its crucial role in heart development and repair [More]
Genetics researchers identify second gene that gives rise to Alagille syndrome

Genetics researchers identify second gene that gives rise to Alagille syndrome

In a finding that may have broader implications for understanding kidney disorders, genetics researchers at The Children's Hospital of Philadelphia have identified a second gene that gives rise to Alagille syndrome. [More]