Publications by authors named "D J Mogul"

Background: Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults.

Methods: Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical trials.

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Article Synopsis
  • Progressive familial intrahepatic cholestasis (PFIC) is an autosomal recessive disorder mainly caused by BSEP deficiency, leading to issues like bile formation disruption and severe itching (pruritus).!* -
  • The MARCH-PFIC study was a phase 3, double-blind, placebo-controlled trial involving participants aged 1-17 from 29 centers across 16 countries, focusing on the effects of the drug maralixibat on different types of PFIC.!* -
  • The study aimed to measure changes in pruritus severity and serum bile acid levels over 26 weeks, specifically looking at outcomes in cohorts with varying forms of PFIC, comparing those treated with maralixibat
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Immune responses to COVID-19 vaccination are attenuated in adult solid organ transplant recipients (SOTRs) and additional vaccine doses are recommended for this population. However, whether COVID-19 mRNA vaccine responses are limited in pediatric SOTRs (pSOTRs) compared to immunocompetent children is unknown. Due to SARS-CoV-2 evolution and mutations that evade neutralizing antibodies, T cells may provide important defense in SOTRs who mount poor humoral responses.

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Background: Neuroendocrine tumors (NETs) are rare epithelial neoplasms that arise most commonly from the gastrointestinal tract. In pediatrics, the most common site of origin is in the appendix, with the liver being the most common site of metastasis. Neuroendocrine tumors arising from the biliary tract are extremely rare.

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Background: Adolescent solid organ transplant recipients (aSOTRs) who received three doses of the COVID-19 mRNA vaccine experience high seroconversion rates and antibody persistence for up to 3 months. Long-term antibody durability beyond this timeframe following three doses of the SARS-CoV-2 mRNA vaccine remains unknown. We describe antibody responses 6 months following the third vaccine dose (D3) of the BNT162b2 mRNA vaccination among aSOTRs.

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