Medical Therapy. Adverse Effects or Lack of Adverse Effects

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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Iatrogenic esophageal dysmotility as a barrier to transplantation in pulmonary arterial hypertension

Michael S. Miller, Shelsey W. Johnson, Alexander R. Opotowsky, Michael J. Landzberg, Nirmal S. Sharma, Hilary J. Goldberg, Alexandra K. Wong, Alison S. Witkin, Josanna Rodriguez-Lopez, Ronald H. Goldstein, Bradley A. Maron, Bradley M. WertheimBrigham and Women’s Hospital, Boston Children’s Hospital and Harvard Medical School. Veterans Affairs Boston Healthcare System. University of Cincinnati School of

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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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Valsartan to Prevent Acquired Pulmonary Vein Stenosis in Pediatric Patients After Total Anomalous Pulmonary Venous Connection Surgery

Jin Shentu, Mingjie Zhang, Zhuoming Xu, Chen Wen, Hao Zhang, Zhongqun Zhu, Huiwen Chen, Guocheng ShiShanghai Children’s Medical Center and Shanghai Jiao Tong University. China Journal of the American Heart AssociationJ Am Heart Assoc 2025; 14: DOI: 10.1161/JAHA.124.036911 AbstractBackground: Recurrent pulmonary vein stenosis (PVS) following surgical repair of total anomalous pulmonary venous connection is associated with poor prognosis. Preclinical studies have shown that use of an angiotensin receptor blocker can attenuate intimal

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Inhaled Epoprostenol in Children With Pediatric Acute Respiratory Distress Syndrome: A Single-Center Retrospective Study

Eleanor J. Scalone, Alan G. Woodruff, Amit K. Saha, John M. Wright, Kristopher L. Dixon, Andora L. Bass, Michael J. Walsh, Michael C. McCroryWake Forest University School of Medicine. University of Pennsylvania Perelman School of Medicine.United States Pediatric PulmonologyPediatr Pulmonol 2025; 60: DOI: 10.1002/ppul.71010 AbstractBackground: Inhaled epoprostenol (iEpo) may improve oxygenation in adults with hypoxic respiratory failure,

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Bosentan as adjunctive therapy in neonates with congenital diaphragmatic hernia-associated pulmonary hypertension: a case series

Aster De Vadder, Lotte Lemloh, Bartolomeo Bo, Lennart Hale, Neil Patel, Andreas Mueller, Florian KipfmuellerChildren’s Hospital, University of Bonn. Royal Hospital for Children. Germany and United Kingdom European Journal of PediatricsEur J Pediatr 2025; 184: DOI: 10.1007/s00431-025-06019-6 AbstractCongenital diaphragmatic hernia (CDH)-associated pulmonary hypertension (PH) is associated with high morbidity and mortality. Pulmonary vasodilative management is challenging

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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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Amniotic fluid stem cell extracellular vesicles as a novel fetal therapy for pulmonary hypoplasia: a review on mechanisms and translational potential

Fabian Doktor, Lina Antounians, Rebeca Lopes Figueira, Kasra Khalaj, Miriam Duci, Augusto ZaniHospital for Sick Children and University of Toronto. Leipzig University.Canada and Germany Stem Cells Translational MedicineStem Cells Transl Med 2025; 14: DOI: 10.1093/stcltm/szae095 AbstractDisruption of developmental processes affecting the fetal lung leads to pulmonary hypoplasia. Pulmonary hypoplasia results from several conditions including congenital diaphragmatic

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