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About 425 results

ALLMedicine™ Alagille Syndrome Center

Research & Reviews  158 results

Macular atrophy in JAG1-related Alagille syndrome: a case series.
https://doi.org/10.1080/13816810.2021.2004432
Ophthalmic Genetics; Paez-Escamilla M, Scanga HL et. al.

Dec 11th, 2021 - : Alagille syndrome (AS) is a multisystem disorder associated with a range of ocular anomalies affecting the anterior and posterior segments. While chorioretinal abnormalities have been reported in Alagille Syndrome, identification of macular dyst...

Maralixibat: First Approval.
https://doi.org/10.1007/s40265-021-01649-0 10.1002/hep4.1244 10.1002/hep.20295 10.1002/hep4.1305 10.1016/S0140-6736(21)01256-3 10.1016/S0618-8278(19)30232-4
Drugs Shirley M

Nov 24th, 2021 - Maralixibat (Livmarli™) is an orally-administered, small-molecule ileal bile acid transporter (IBAT) inhibitor being developed by Mirum Pharmaceuticals for the treatment of rare cholestatic liver diseases including Alagille syndrome (ALGS), progre...

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
https://clinicaltrials.gov/ct2/show/NCT02047318

Nov 19th, 2021 - The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol...

Infantile Scoliosis
https://emedicine.medscape.com/article/1259899-overview

Nov 12th, 2021 - Practice Essentials The term scoliosis is derived from the Greek word skolios ("twisted") and refers to a sideward (right or left) curve in the spine. Scoliosis is not a simple curve to one side but, in fact, is a more complex three-dimensional de...

Infantile Scoliosis
http://emedicine.medscape.com/article/1259899-overview

Nov 12th, 2021 - Practice Essentials The term scoliosis is derived from the Greek word skolios ("twisted") and refers to a sideward (right or left) curve in the spine. Scoliosis is not a simple curve to one side but, in fact, is a more complex three-dimensional de...

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Drugs  1 results see all →

Clinicaltrials.gov  5 results

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
https://clinicaltrials.gov/ct2/show/NCT02047318

Nov 19th, 2021 - The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol...

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
https://clinicaltrials.gov/ct2/show/NCT04530994

Oct 20th, 2021 - The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
https://clinicaltrials.gov/ct2/show/NCT02057692

Mar 26th, 2019 - The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical...

Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
https://clinicaltrials.gov/ct2/show/NCT01515631

Sep 28th, 2018 - The investigators hope to learn the degree and characteristics of pulmonary artery stenosis in Alagille Syndrome. We also hope to learn the effect of this pulmonary artery stenosis on the right ventricle in patients with Alagille Syndrome. This in...

Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
https://clinicaltrials.gov/ct2/show/NCT00001642

Mar 4th, 2008 - The goal of the project is to identify and clone the gene(s) responsible for the Alagille Syndrome (AGS) by a positional cloning approach. The first step towards this goal is to define the smallest genomic candidate region for AGS at 20p12 and to ...

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News  5 results

Mirum's Oral Drug Becomes First US-Approved Alagille Syndrome Therapy
https://www.medscape.com/viewarticle/959976

Sep 29th, 2021 - Mirum Pharmaceuticals Inc said its drug became the first U.S. approved therapy for treating itching in patients with rare liver disorder Alagille syndrome (ALGS), and would be available for distribution immediately. Shares of the company were brie...

FDA OKs First Drug for Symptoms of Rare Liver Disease in Kids
https://www.medpagetoday.com/gastroenterology/generalhepatology/93673

Jul 21st, 2021 - The FDA approved odevixibat (Bylvay) for treating young children with intense itching caused by progressive familial intrahepatic cholestasis (PFIC), manufacturer Albireo Pharma announced on Tuesday. Odevixibat becomes the first therapy for the ra...

Progress in Fatty Liver and Pediatric Liver Disease
https://www.staging.medscape.com/viewarticle/886923

Oct 11th, 2017 - WASHINGTON, DC — Advances in the treatment of nonalcoholic steatohepatitis (NASH) — including results from a phase 2 trial of a liver-targeted selective inhibitor of acetyl-CoA carboxylase — will be in the spotlight here at The Liver Meeting 2017:...

Progress in Fatty Liver and Pediatric Liver Disease
https://www.medscape.com/viewarticle/886923

Oct 11th, 2017 - WASHINGTON, DC — Advances in the treatment of nonalcoholic steatohepatitis (NASH) — including results from a phase 2 trial of a liver-targeted selective inhibitor of acetyl-CoA carboxylase — will be in the spotlight here at The Liver Meeting 2017:...

Progress Noted in Treatment of Several Common Liver Diseases
https://www.medscape.com/viewarticle/861425_6

Apr 8th, 2016 - Intrahepatic Cholestasis Neonatal cholestasis results from a heterogeneous group of intrahepatic disorders caused by mutations in several genes.[28] Recent advances in the understanding of the molecular basis of genetic cholestatic syndromes, such...

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Patient Education  1 results see all →