Mirum ALGS Series: Jocelyn скачать в хорошем качестве

Mirum ALGS Series: Jocelyn

Mirum is committed to creating life-changing therapies for patients like Jocelyn. For more info about clinical studies and treatment availability, please contact us at [email protected], or visit algseap.com for updates and to sign up for notifications about the Expanded Access Program for children with ALGS. ALGS is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and leads to progressive liver disease that ultimately requires liver transplantation in 15% to 47% of patients. Signs and symptoms arising from liver damage in ALGS may include jaundice, pruritus and xanthomas. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life. Children with ALGS experience a markedly impaired quality of life largely due to the intense pruritus and associated skin lesions and disruptions in sleep and mood. There remains a substantial unmet medical need for therapeutic options for ALGS, as partial external biliary diversion and liver transplantation are the only options available for these patients.