Medical Therapy. Pharmacokinetics and Pharmacology Archives

Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy

Noor Ul Ain, Armaan Saith, Audrey Ruan, Ruhua Yang, Aaron Burton, Pramod K. MistryYale School of Medicine and Yale New Haven Hospital.United States Frontiers in MedicineFront Med 2025; 12: DOI: 10.3389/fmed.2025.1535099 AbstractIntroduction: Gaucher disease (GD), a lysosomal storage disorder, results from the accumulation of glycosphingolipids due to deficient lysosomal glucocerebrosidase activity. This pathological accumulation triggers immune activation, […]

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Trametinib as a targeted treatment in cardiac and lymphatic presentations of Noonan syndrome

Isabel De Brouchoven, Juan Lorand, Léon Bofferding, Arthur Sorlin, An Van Damme, Olivier DanhaiveSaint-Luc University Hospital,. Luxembourg Hospital Center. Laboratoire National de Santé. University of California San Francisco.Belgium, Luxembourg and United States Frontiers in PediatricsFront Pediatr 2025; 13: DOI: 10.3389/fped.2025.1475143 AbstractIntroduction: Rare pathogenic variants in the PTPN11, KRAS, SOS1 and RAF1 genes are the main molecular causes of Noonan syndrome (NS).

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Macitentan: Pediatric First Approval

Susan J. KeamSpringer Nature.New Zealand Paediatric DrugsPaediatr Drugs 2025; DOI: 10.1007/s40272-025-00692-8 AbstractMacitentan (Opsumit®), an endothelin receptor antagonist (ERA) developed by Johnson & Johnson, is well established worldwide as monotherapy or combination therapy for the long-term treatment of pulmonary arterial hypertension (PAH). In September 2024, based on phase 3 clinical data in patients aged < 18 years,

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Mechanism of action of aloperine in the treatment of pulmonary arterial hypertension based on network pharmacology and molecular docking methods

Yanrong Wang, Baolan Yan, Pengsheng Ma, Ru Zhou, Fang ZhaoGeneral Hospital of Ningxia Medical University and Ningxia Medical University. China Herz Cardiovascular DiseaseHerz 2025; DOI: 10.1007/s00059-025-05295-0 AbstractBackground: Pulmonary arterial hypertension is a severe pulmonary vascular disease, marked by high mortality and substantial treatment costs, underscoring the urgent need for the exploration of traditional Chinese medicine as a

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Targeting Lung Heme Iron by Aerosol Hemopexin Administration in Sickle Cell Disease Pulmonary Hypertension

Melissa Lucero, Christina Lisk, Francesca Cendali, Delaney Swindle, Saini Setua, Kiruphagaran Thangaraju, David I. Pak, Quinton O’Boyle, Shuwei Lu, Robert Tolson, Seth Zaeske, Nishant Rana, Saqib Khan, Natalie Westover, Pavel Davizon Castillo, Gemlyn George, Kathryn Hassel, Rachelle Nuss, Nathan Brinkman, Thomas Gentinetta, Andre F. Palmer, Angelo D’Alessandro, Paul W. Buelher, David C. IrwinUniversity of Colorado,

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Safety and Efficacy of Selexipag for Pediatric Pulmonary Arterial Hypertension in Japanese Patients – An Open-Label Phase 2 Study

Toru Iwasa, Ryo Inuzuka, Hiroshi Ono, Yuichiro Sugitani, Hirokuni Yamazawa, Chihiro Hiraishi, Naoki Shiota, Shinichi Tanaka, Chieko Yamamoto, Ken-ichi Kurosaki, Masaru Miura, Satoshi YasukochiNational Cerebral and Cardiovascular Center. University of Tokyo. National Center for Child Health and Development. Japan Community Health Care Organization Kyushu Hospital. Hokkaido University. Nippon Shinyaku Company. Tokyo Metropolitan Children’s Medical Center.

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The Role of Nitric oxide in the sweep gas for patients receiving Extracorporeal Membrane Oxygenation or Cardiopulmonary Bypass

Roberto Chiletti, Sophie H. Fincher, Stephen B. Horton, Giles J. Peek, Paul Checchia, Warwick ButtRoyal Children’s Hospital and University of Melbourne. University of Florida. Texas Children’s Hospital and the Baylor College of Medicine. Australia and United States Canadian Journal of CardiologyCan J Cardiol 2024; DOI: 10.1016/j.cjca.2024.12.027 AbstractNitric oxide (NO) was proclaimed the 1992 “molecule of the

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Population pharmacokinetics of selexipag for dose selection and confirmation in pediatric patients with pulmonary arterial hypertension

Lene Nygaard Axelsen, Anne Kümmel, Juan Jose Perez Ruixo, Alberto RussuActelion Pharmaceuticals and Johnson & Johnson, Allschwil. Janssen-Cilag S.p.A.Switzerland, Spain and Italy Clinical Pharmacology and Therapeutics: Pharmacometrics and Systems PharmacologyCPT Pharmacometrics Syst Pharmacol 2024;DOI: 10.1002/psp4.13231 AbstractSelexipag is an oral selective prostacyclin receptor agonist approved for the treatment of pulmonary arterial hypertension (PAH) in adults. To date,

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Oxygen saturation targets in neonatal care: A narrative review

Tri C. Nguyen, Rajeshwari Madappa, Heather M. Siefkes, Michelle J. Lim, Kanya Mysore Siddegowda, Satyan LakshminrusimhaKaiser Permanente North California. SIGMA Hospital. University of California Davis Children’s Hospital.United States Early Human DevelopmentEarly Hum Dev 2024; 199:DOI: 10.1016/j.earlhumdev.2024.106134 AbstractOptimal oxygenation requires the delivery of oxygen to meet tissue metabolic demands while minimizing hypoxic pulmonary vasoconstriction and oxygen toxicity.

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