Medical Therapy. Adverse Effects or Lack of Adverse Effects

Macitentan in Children with Pulmonary Arterial Hypertension: A Multicenter Experience

A. Rodriguez Ogando, A. Sabaté-Rotés, A. Moya Bonora, L. Albert de la Torre, J. M. Espín López, C. Labrandero de Lera, Maria Jesus Del Cerro MarinGregorio Marañon General University Hospital. Hospital Materno Infantil Gregorio Marañon. Vall d’Hebron University Hospital. La Fe University Hospital. 12 de Octubre University Hospital. Virgen dee la Arrixaca University Hospital. La […]

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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 »

Role of phosphodiesterase-3 inhibitor in cardiorespiratory fitness and functional class of patients with pulmonary hypertension: A randomized, double-blind, placebo-controlled trial

Sefri N. Sofia, Udin Bahrudin, Ilham Uddin, Muhammad A. Sobirin, Erna Setiawati, Galuh Hardaningsih, Kevin C. Tjandra, Edward K. S. LimijadiUniversitas Diponegoro. Dr. Kariadi Hospital. Indonesia Narra JNarra J 2025; 5: DOI: 10.52225/narra.v5i1.1301 AbstractMany patients with acyanotic shunt congenital heart disease (CHD) are diagnosed only in adulthood, by which time pulmonary hypertension (PH) has developed, impairing

Role of phosphodiesterase-3 inhibitor in cardiorespiratory fitness and functional class of patients with pulmonary hypertension: A randomized, double-blind, placebo-controlled trial Read More »

Inhibiting Serotonin Synthesis for the Treatment of Pulmonary Arterial Hypertension

Georg Hansmann, Michael BaderFriedrich-Alexander-University Erlangen-Nürnberg. Trypto Therapeutics. Charité-Universitätsmedizin. University of LübeckGermany Pulmonary CirculationPulm Circ 2025; 15: DOI: 10.1002/pul2.70100 AbstractAbstract Not Available CategoryMedical Therapy. Efficacy or Lack of EfficacyMedical Therapy. Adverse Effects or Lack of Adverse Effects Age Focus: No Age-Related Focus Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication Article Access Free

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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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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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