Medical Therapy. Efficacy or Lack of Efficacy Archives

The sonic hedgehog signaling inhibitor cyclopamine improves pulmonary arterial hypertension via regulating the bone morphogenetic protein receptor 2 pathway

Youpeng Jin, Fei Mao, Xuehui Wang, Jie Zhang, Yanting Gao, Youfei FanShandong Provincial Hospital, First Affiliated Hospital and Shandong First Medical University.China Scientific ReportsSci Rep 2025; 15: DOI: 10.1038/s41598-025-97627-7 AbstractPulmonary arterial hypertension (PAH) is a severe and progressive disease with hallmarks of pulmonary vascular remodeling and bone morphogenetic protein receptor 2 (BMPR2) mutation. Recent studies indicate […]

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Novel Use of Drug-Coated Balloon Angioplasty to Treat Pulmonary Vein Stenosis

Saloni Agrawal, Kali A. Hopkins, Ali N. Zaidi, Barry LoveMount Sinai Hospital. Mount Sinai Fuster Heart Hospital.United States Journal of the American College of Cardiology Case ReportsJACC Case Rep 2024; 29: DOI: 10.1016/j.jaccas.2024.102613 AbstractPulmonary vein stenosis is challenging to treat due to high rate of recurrence. Multiple interventions exist but are limited by high rates of

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Paclitaxel-coated drug-eluting balloon for pulmonary vein stenosis after repair of total anomalous pulmonary venous return with asplenia

Hiroshi Ono, Ryo Mafune, Taiyu Hayashi, Yasushi Misaki, Yukihiro Kaneko, Jumpei Saito, Miki Nagai, Hitoshi KatoNational Medical Center for Child Health and Development. Japan Journal of Cardiology CasesJ Cardiol Cases 2020; 22: 107-109DOI: 10.1016/j.jccase.2020.05.008 AbstractWe used a paclitaxel-coated drug-eluting balloon (DEB) for pulmonary vein stenosis (PVS) after repair of total anomalous pulmonary venous return with asplenia.

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Paclitaxel drug-eluting stent placement for pulmonary vein stenosis as a bridge to heart-lung transplantation

Andreea Dragulescu, Olivier Ghez, Jacques Quilici, Alain FraisseCentre Hospitalo-Universitaire La Timone.France Pediatric CardiologyPediatr Cardiol 2009; 30: 1169-1171DOI: 10.1007/s00246-009-9511-5 AbstractCongenital pulmonary vein stenosis (PVS) presents as an isolated lesion or in association with other congenital heart anomalies. The most extensive forms of the disease are uniformly fatal because neither surgical repair nor transcatheter therapy results in long-term

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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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Nasopharyngeal ventilation: an answer to the conundrum of severe nasal injury in a neonate

Bhabesh Kant Chowdhry, Arnab Ghorui, Richie Dalai, Rameshwar PrasadAll India Institute of Medical Sciences.India British Medical Journals Case ReportsBMJ Case Rep 2025; 18:DOI: 10.1136/bcr-2025-264797 AbstractA term neonate with a stormy perinatal course, requiring prolonged mechanical ventilation, due to congenital pneumonia, complicated by secondary pulmonary arterial hypertension and prolonged non-invasive respiratory support, presented to us in the

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Respiratory Support Strategies for Surgical Neonates: A Review

Piero Alberti, Niyi Ade-Ajayi, Anne GreenoughKing’s College London.United Kingdom ChildrenChildren 2025; 12: DOI: 10.3390/children12030273 AbstractNeonates with congenital conditions which require surgical management frequently experience respiratory distress. This review discusses the management of pulmonary complications and the respiratory support strategies for four conditions: oesophageal atresia-tracheoesophageal fistula (OA-TOF), congenital diaphragmatic hernia (CDH), congenital lung malformations (CLM), and anterior

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Intraamniotic Vitamin D Preserves Lung Development and Prevents Pulmonary Hypertension in Experimental Bronchopulmonary Dysplasia due to Intraamniotic sFlt-1

Michael W. Cookson, Tania Gonzalez, Elisa M. Bye, Greg Seedorf, Sarah Ellor, Brad J. Smith, James C. Fleet, Erica W. MandellUniversity of Colorado, Anschutz School of Medicine and Children’s Hospital Colorado. University of Texas.United States American Journal of Physiology Lung Cellular and Molecular PhysiologyAm J Physiol Lung Cell Mol Physiol 2025; DOI: 10.1152/ajplung.00409.2024 AbstractPreterm infants born

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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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Clinical characteristics and impact of exchange transfusion in infant pertussis with extreme leukocytosis

Yuanyuan Wu, Chuan GanSecond Affiliated Hospital of Chongqing Medical University, Children’s Hospital of Chongqing Medical University and Chongqing Medical University. Chongqing Key Laboratory of Child Rare Diseases in Infection and Immunity.China Italian Journal of PediatricsItal J Pediatr 2025; 51: DOI: 10.1186/s13052-025-01933-9 AbstractBackground: Extreme leukocytosis in pertussis is a rare condition, and without effective interventions to reduce white

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