Medical Therapy. Pharmacokinetics and Pharmacology

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 »

Oxygen Saturation Targeting in the Neonatal Intensive Care Unit

Faeq Almudares, Bheru Gandhi, Jonathan Davies, Xanthi Couroucli, Natalie Villafranco, Nidhy Paulose Varghese, Milenka Cuevas Guaman, Charleta Guillory, Binoy ShivannaBaylor College of Medicine.United States Journal of Clinical MedicineJ Clin Med 2025; 14: DOI: 10.3390/jcm14113975 AbstractOxygen (O2) is vital for cellular development, function, proliferation, and repair, underscoring its critical role in organogenesis. Both hypoxia (reduced tissue O2)

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Comparative study between nebulized and intravenous magnesium sulfate for treatment of persistent pulmonary hypertension in neonates

Nashwa Farouk Mohamed, Osama Abu El Fetouch Zaki El Feky, Heba Morsy Saad El Din El Ganady, Walid Abd Elatif Abd El HalimBenha University.Egypt Journal of Neonatal-Perinatal MedicineJ Neonatal Perinatal Med 2025; 18: 246-254DOI: 10.1177/19345798251325510 AbstractBackground: Persistent pulmonary hypertension of the newborn (PPHN) is a life-threatening condition resulting from elevated pulmonary vascular resistance, causing severe hypoxemia.

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Performance of oral Bosentan-loaded SNEDDS and S-SNEDDS tablets: Biodistribution in mice, echocardiography and histology studies in pulmonary arterial hypertension rat model

Duygu Yilmaz Usta, Seval Olgac, Murside Ayse Demirel, Serdar Kula, Cigdem Elmas, Perihan Sezginer, Akif Kavgaci, Zeynep Safak TeksinGazi University. Alanya Alaadin Keykubat University. Turkey European Journal of Pharmaceutics and BiopharmaceuticsEur J Pharma Biopharma 2025; DOI: 10.1016/j.ejpb.2025.114725 AbstractBosentan monohydrate (BOS) is the most preferred molecule for treating the rare pulmonary arterial hypertension (PAH) disease. BOS shows

Performance of oral Bosentan-loaded SNEDDS and S-SNEDDS tablets: Biodistribution in mice, echocardiography and histology studies in pulmonary arterial hypertension rat model Read More »

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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