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About 545 results

ALLMedicine™ Alagille Syndrome Center

Research & Reviews  191 results

Pathogenic Novel Heterozygous Variant c.1076c>T p. (Ser359Phe) chr1: 120512166 in NOTCH...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9552858
The American Journal of Case Reports; Uddin MS, Al Fulayyih S et. al.

Oct 7th, 2022 - BACKGROUND Alagille syndrome (ALGS) is a multisystem hereditary illness with a dominant pattern and partial penetrance. Multiple organ abnormalities can be caused by mutations in the Jagged canonical Notch ligand 1 (JAG1) gene. Notch receptor 2 (N...

Health care resource utilization by patients with Alagille syndrome.
https://doi.org/10.1016/j.jpeds.2022.09.033
The Journal of Pediatrics; Ebel NH, Goldstein A et. al.

Oct 1st, 2022 - To assess and characterize health care resource utilization (HRU) in children with the rare, genetic, multisystem disorder, Alagille syndrome. This retrospective analysis reviewed commercially insured and Medicaid-insured claims from October 1, 20...

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
https://clinicaltrials.gov/ct2/show/NCT05543174

Sep 16th, 2022 - The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work...

Maralixibat treatment response in Alagille syndrome is associated with improved health-...
https://doi.org/10.1016/j.jpeds.2022.09.001
The Journal of Pediatrics; Kamath BM, Goldstein A et. al.

Sep 13th, 2022 - The objective of this study was to assess the impact of treatment response to the ileal bile acid transporter inhibitor maralixibat on health-related quality of life (HRQoL) in children with Alagille syndrome. This analysis used data from the ICON...

Surgical repair of peripheral pulmonary artery stenosis: A 2-decade experience with 145...
https://doi.org/10.1016/j.jtcvs.2022.07.037
The Journal of Thoracic and Cardiovascular Surgery; Felmly LM, Mainwaring RD et. al.

Sep 11th, 2022 - Peripheral pulmonary artery stenosis (PPAS) is a relatively rare form of congenital heart disease often associated with Williams syndrome, Alagille syndrome, and elastin arteriopathy. This disease is characterized by stenoses at nearly all lobar a...

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Drugs  1 results see all →

Clinicaltrials.gov  13 results

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
https://clinicaltrials.gov/ct2/show/NCT05543174

Sep 16th, 2022 - The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work...

Atorvastatin Therapy on Xanthoma in Alagille Syndrome
https://clinicaltrials.gov/ct2/show/NCT05488067

Aug 4th, 2022 - Alagille syndrome (ALGS, OMIM 118450) is an important cause of chronic cholestasis in children, and the incidence rate is about 1:30000~1:50000. Most patients with ALGS have hypercholesterolemia. In severe cases, multiple xanthomas can be seen, an...

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
https://clinicaltrials.gov/ct2/show/NCT04674761

Mar 31st, 2022 - Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
https://clinicaltrials.gov/ct2/show/NCT05035030

Mar 10th, 2022 - Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

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News  11 results

Entyvio for Kid IBD; Phase III Win in PFIC; Drug Flops for Eosinophilic Esophagitis
https://www.medpagetoday.com/gastroenterology/generalgastroenterology/101455

Oct 27th, 2022 - Vedolizumab (Entyvio) was both safe and effective at inducing clinical remission for kids with inflammatory bowel disease (IBD), especially for those with ulcerative colitis, according to the 14-week VEDOKIDS study. This treatment should be consid...

Predicting Crohn's Relapse; Sex After Hernia Repair; Bacteria and Anti-TNF Response
https://www.medpagetoday.com/gastroenterology/generalgastroenterology/100505

Sep 1st, 2022 - Distinct blood protein profiles may help predict clinical relapse in Crohn's disease patients who stop tumor necrosis factor (TNF) inhibitors, and could help pave the way for non-invasive biomarkers to guide anti-TNF withdrawal. (Gut) What's bette...

IBAT Inhibitors: The Medication That Patients With Pruritus Were Itching to Try
https://www.medscape.com/viewarticle/974182

May 23rd, 2022 - Pruritus is a prime clinical expression of cholestatic diseases in children and adults. In my clinical experience, there was no more poignant depiction of the ravages of cholestatic pruritus than that of an 11-year-old girl with Alagille syndrome ...

YouTube Bans Anti-Vaxxers; UV Disinfection Danger; Beijing's Olympics Rules
https://www.medpagetoday.com/infectiousdisease/covid19/94782

Sep 30th, 2021 - Note that some links may require registration or subscription. Anti-vaccine videos will no longer have a home on YouTube. (Washington Post) EHR data suggested that long COVID affected one in three people who had been infected, and lingering sympto...

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...

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Patient Education  2 results see all →