Alagille Syndrome (ALGS) is a genetic disorder that ... This syndrome is often diagnosed in infants and children, but its manifestations can vary widely, making it a complex condition to manage.

Sarah Love was a little taken aback when a stranger approached her and made an unusual comment about her newborn son's skin color. But it saved little Lincoln's - revealing her boy had a silent ...

In unusual circumstances, Ipsen has been given the green light for its odevixibat drug for severe itching (pruritus) in patients with rare liver disease Alagille syndrome (ALGS) in the EU – for ...

Founded in 2018, Mirum Pharmaceuticals, Inc. (MIRM) is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved ...

are suitable [ID6291] Technology appraisal guidance TBC Somapacitan for treating growth hormone deficiency in children [ID6178] Technology appraisal guidance TBC Spesolimab for preventing generalised ...

It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older.

Distinct protein profiles linked to inflammation and angiogenesis found in children with Long COVID, paving the way for ...

Down syndrome affects around 1 in 800 new births and is caused by an extra third copy of chromosome 21. About half of children with Down syndrome have hearing loss due to otitis media with ...

Marcelo was diagnosed with Treacher Collins syndrome, a rare condition affecting facial bone structure and cartilage. The ...