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Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Neurology
January 2025
The Dubowitz Neuromuscular Centre, Developmental Neurosciences Department, University College London, Great Ormond Street Institute of Child Health, United Kingdom.
Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.
View Article and Find Full Text PDFIron deficiency in acute coronary syndromes: prevalence and prognostic impact.
Porto Biomed J
January 2025
Rua Camilo Castelo Branco, Cardiology Department, São Bernardo Hospital, Setúbal Hospital Centre, Setúbal, Portugal.
Introduction And Objectives: Iron deficiency (ID) is a well-known prognostic marker in heart failure (HF), independent of anemia. However, its impact in acute coronary syndromes (ACSs) is not well established.
Methods: This is a retrospective analysis of patients admitted with ACS from January to December 2019.
Brainstem C1 neurons mediate heart failure decompensation and mortality during acute salt loading.
Cardiovasc Res
December 2024
Laboratory of Cardiorespiratory Control, Department of Physiology, Pontificia Universidad Católica de Chile, Av. Libertador Bernardo O'Higgins 340, Santiago 8331150, Chile.
Aims: Heart failure (HF) is an emerging epidemic worldwide. Despite advances in treatment, the morbidity and mortality rate of HF remain high, and the global prevalence continues to rise. Common clinical features of HF include cardiac sympathoexcitation, disordered breathing, and kidney dysfunction; kidney dysfunction strongly contributes to sodium retention and fluid overload, leading to poor outcomes of HF patients.
View Article and Find Full Text PDFSpontaneous Tumor Lysis Syndrome in a Thoracic Burkitt Lymphoma: A Case Report.
Cureus
November 2024
Hematology, Centro Hospitalar de Setúbal, Hospital de São Bernardo, Setúbal, PRT.
Burkitt lymphoma has a high proliferation rate and a significant risk of tumor lysis syndrome. Risk stratification and early identification are imperative since it is an oncological emergency. We report the case of a 20-year-old woman, without relevant past medical history, admitted to the Emergency Department with a three-week history of fatigue, chest discomfort, productive cough, night sweats, myalgia, odynophagia, and holocranial headache.
View Article and Find Full Text PDFIncidence and predictors of gastrointestinal hemorrhage following mesenteric revascularization.
J Vasc Surg
November 2024
Division of Vascular and Endovascular Surgery, Mayo Clinic, Rochester, MN. Electronic address:
Objective: Postoperative gastrointestinal hemorrhage (GIH) following mesenteric revascularization when performed either openly (OR) or endovascularly (ER) has been clinically observed but not reported. The aim of the study is to assess the incidence and predictors of GIH in patients undergoing mesenteric revascularization.
Methods: This was a single-center retrospective review of consecutive patients treated with open or endovascular mesenteric revascularization from 2009 to 2019.
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