Medical Therapy. Pharmacokinetics and Pharmacology

Macitentan vs Standard of Care in Pediatric Pulmonary Arterial Hypertension (TOMORROW): A Randomized Clinical Trial

Rolf M. F. Berger, D. Dunbar Ivy, Julian I. Borissoff, Sofija Cerovic, Dénes Csonka, Tatiana Remenová, Dominik Richard, Matthieu Villeneuve, Maurice BeghettiBeatrix Children’s Hospital, University Medical Center Groningen and University of Groningen. Children’s Hospital Colorado. Johnson & Johnson. University Hospitals of Geneva.Netherlands, United States and Switzerland Journal of PediatricsJ Pediatr 2026; DOI: 10.1016/j.jpeds.2026.115057 AbstractObjectives: To evaluate pharmacokinetics […]

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Development of a Robust, Rapid and Reliable Tandem Mass Spectrometry Method for the Measurement of Sildenafil, Bosentan and their Major Metabolites

Mohammad Ahmad Bik, Duygu Eryavuz, Onmaz, Karem Mazin Kamil Gharab, Fadime Karaman, Sedat Abusoglu, Abdullah Sivrikaya, Ali UnluSelcuk University. Mustansiriyah University.Turkey and Iraq Indian Journal of Clinical BiochemistryIndian J Clin Biochem 2026; 41: 113-119DOI: 10.1007/s12291-024-01215-x AbstractPulmonary arterial hypertension (PAH) is a lethal, progressive disease with a complex pathogenesis. Bosentan, a dual endothelin receptor antagonist, and sildenafil,

Development of a Robust, Rapid and Reliable Tandem Mass Spectrometry Method for the Measurement of Sildenafil, Bosentan and their Major Metabolites Read More »

Safety of a novel portable inhaled nitric oxide generator for therapy of persistent pulmonary hypertension in neonates: a case series study

Yao Zhu, Jiongzhi He, Miner Cai, Lijun Wen, Minxu LiDongguan Maternal and Child Health Care Hospital. First Affiliated Hospital of Jinan University.China Translational PediatricsTransl Pediatr 2025; 14: 3305-3317DOI: 10.21037/tp-2025-535 AbstractBackground: Persistent pulmonary hypertension of the newborn (PPHN) is a life-threatening emergency in neonatal intensive care. Traditional treatments such as cylinder-based nitric oxide (NO) delivery systems rely on

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A prospective, multicenter, open-label, single-arm Phase 2 study to investigate the pharmacokinetics, safety, tolerability, and exploratory efficacy of selexipag in children with pulmonary arterial hypertension

Maurice Beghetti, Lene Nygaard Axelsen, Julian I. Borissoff, Mahdi Farhan, Simon Grill, Sining Leng, Alberto Russu, Catherine Lesage, Tatiana Remeňová, Shu-Fang Hsu Schmitz, Shahin MoledinaUniversity Hospitals of Geneva. Johnson & Johnson. Great Ormond Street Hospital. Switzerland and United Kingdom ChestChest 2025; DOI: 10.1016/j.chest.2025.12.013 AbstractBackground: Selexipag is an oral selective prostacyclin receptor agonist approved for treating pulmonary arterial

A prospective, multicenter, open-label, single-arm Phase 2 study to investigate the pharmacokinetics, safety, tolerability, and exploratory efficacy of selexipag in children with pulmonary arterial hypertension Read More »

Preparation of 18β-Glycyrrhetinic Acid Liposome and Its Therapeutic Effect on Pulmonary Arterial Hypertension

Yanmin Pei, Meidong Si, Xuemei Ma, Siyun Liu, Fang Zhao, Ru ZhouNingxia Medical University and General Hospital of Ningxia Medical University.China Drug Design, Development and TherapyDrug Des Dev Ther 2025; 19: 11119-11144DOI: 10.2147/DDDT.S547530 AbstractPurpose: 18β-Glycyrrhetinic acid liposomes (18β-GA-Lips) were developed to enhance lung-specific drug delivery and optimize the therapeutic management of pulmonary arterial hypertension (PAH).Methods: 18β-GA-Lips of varying

Preparation of 18β-Glycyrrhetinic Acid Liposome and Its Therapeutic Effect on Pulmonary Arterial Hypertension Read More »

Systemic Sirolimus Monitoring in Pediatric Pulmonary Vein Stenosis

Heather Meluskey, Bridget Blowey, Anna B. O’Brien, Michael L. O’Byrne, Jonathan J. Rome, David B. Frank, Catherine M. Avitabile, Mudit Gupta, Jessica Tang, Kimberly L. Butler, Constantine D. Mavroudis, Stephanie Fuller, Ryan CallahanChildren’s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania. Dell School of Medicine at the University of Texas.

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Nebulized Treprostinil in Mechanically Ventilated Children

Sarah P. Cohen, Diane Paulus, Ashley Hattie, Beth Malehorn, Mary Irmen, Robert GajarskiOhio State University and Nationwide Children’s Hospital. United States Pediatric PulmonologyPediatr Pulmonol 2025; 60: DOI: 10.1002/ppul.71278 AbstractAbstract Not Available CategoryMedical Therapy. Efficacy or Lack of EfficacyMedical Therapy. Adverse Effects or Lack of Adverse EffectsMedical Therapy. Pharmacokinetics and Pharmacology Age Focus: Pediatric Pulmonary Vascular Disease

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Enhancing Drug Development for Paediatric Pulmonary Hypertension-An Integrative Perspective

Steven H. Abman, Sylvia M. Nikkho, Rolf M. F. Berger, Maria Jesus del Cerro, Eric D. Austin, Maurice Beghetti, Dunbar Ivy, Megan Griffiths, Anne Hilgendorff, Steven H. Kawut, Usha S. Krishnan, Mary P. Mullen, Shahin Moledina, Bernard Thébaud, Norman StockbridgeUniversity of Colorado School of Medicine and Children’s Hospital Colorado. Bayer AG. Beatrix Children’s Hospital, University

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Transferrin Purification, Biophysical Characterization, and Lung Biodistribution in Sickle Cell Disease Mice

Shuwei Lu, Mohd A. Khan, Saini Setua, Quintin O’Boyle, Kiruphagaran Thangaraju, Pedro Cabrales, Delaney C. Swindle, David C. Irwin, Paul W. Buehler, Andre F. PalmerOhio State University. University of Maryland School of Medicine. University of California San Diego. University of Colorado, Anschutz Medical Campus. United States Biotechnology and BioengineeringBiotechnol Bioeng 2025; DOI: 10.1002/bit.70012 AbstractPlasma transferrin (Tf)

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Clinical Study to Evaluate the Possible Efficacy and Safety of L-Arginine in Children with Sickle Cell Disease and Increased Tricuspid Regurgitant Jet Velocity: a Randomized Controlled Trial

Dalia A. Gomaa, Sahar M. El‑Haggar, Mohamed R. El‑Shanshory, Osama El‑Razaky, Dalia R. El‑AffyTanta University. Egypt Paediatric DrugsPaediatr Drugs 2025; DOI: 10.1007/s40272-025-00701-w AbstractBackground: Pulmonary hypertension (PH) is a common chronic complication of sickle cell disease (SCD), and patients at risk for PH can be identified by measuring tricuspid regurgitant jet velocity (TRJV). We looked for the possible

Clinical Study to Evaluate the Possible Efficacy and Safety of L-Arginine in Children with Sickle Cell Disease and Increased Tricuspid Regurgitant Jet Velocity: a Randomized Controlled Trial Read More »

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