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Fenfluramine in the treatment of Dravet syndrome: Results of a third randomized, placebo-controlled clinical trial

Joseph Sullivan, Lieven Lagae, J. Helen Cross, Orrin Devinsky, Renzo Guerrini, Kelly G. Knupp, Linda Laux, Marina Nikanorova, Tilman Polster, Dinesh Talwar, Berten Ceulemans, Rima Nabbout, Gail M. Farfel, Bradley S. Galer, Arnaold R. Gammaitoni, Michael Lock, Anupam Agarwal, Ingrid E. Scheffer, The FAiRE DS Study GroupUniversity of California San Francisco. University of Leuven. Great […]

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Intra-amniotic sildenafil treatment improves lung blood flow and pulmonary hypertension in congenital diaphragmatic hernia rats

Shiho Yoshida, Alexander M. Kreger, George K. Gittes UPMC Children’s Hospital of Pittsburgh and University of Pittsburgh School of Medicine.United States Frontiers in Bioengineering and BiotechnologyFront Bioeng Biotechnol 2023; DOI: 10.3389/fbioe.2023.1195623 AbstractPulmonary hypertension associated with congenital diaphragmatic hernia (CDH) is a critical factor in determining prognosis. We propose that intra-amniotic sildenafil administration is an effective prenatal therapy for

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Measurement of Physical Activity by Actigraphy in Infants and Young Children with Pulmonary Arterial Hypertension

Catherine M. Avitabile, Delphine Yung, Stephanie Handler, Rachel K. Hopper, Jeff Fineman, Grace Freire, Nidhy Varghese, Mary Mullen, Usha S. Krishnan, Eric Austin, Lori Silveira, D. Dunbar IvyChildren’s Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine. University of Washington School of Medicine. Medical College of Wisconsin. Stanford University School of Medicine. University

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Recent Advances in Pediatric Pulmonary Hypertension: Implications for Diagnosis and Treatment

Rachel T. Sullivan, J. Usha Raj, Eric D. AustinVanderbilt University Medical Center and Monroe Carrell Jr Children’s Hospital. University of Illinois at Chicago.United States Clinical TherapyClin Ther 2023; DOI: 10.1016/j.clinthera.2023.07.001 AbstractPurpose: Pediatric pulmonary hypertension (PH) is a condition characterized by elevated pulmonary arterial pressure, which has the potential to be life-limiting. The etiology of pediatric PH varies.

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Spontaneous neonatal pulmonary arterial thrombosis – cases, mechanisms, and literature review

Ali Abdelhafiz, Alexis L. Benscoter, Sarosh P. BatlivalaCincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine.United States Cardiology in the YoungCardiol Young 2023; DOI: 10.1017/S1047951123002639 AbstractSpontaneous pulmonary artery thrombosis in neonates is rare and can be life-threatening. Clinical presentation may mimic pulmonary hypertension or CHD. Further, not all children present with identifiable risk

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Vasopressin versus epinephrine during neonatal cardiopulmonary resuscitation of asphyxiated post-transitional piglets

Megan O’Reilly, Tze-Fun Lee, Po-Yin Cheung, Georg M. SchmolzerRoyal Alexandra Hospital and University of Alberta.Canada Resuscitation PlusResus Plus 2023; 15DOI: 10.1016/j.resplu.2023.100427 AbstractBackground: Epinephrine is currently the only recommended cardio-resuscitative medication for use in neonatal cardiopulmonary resuscitation (CPR), as per the consensus of science and treatment recommendations. An alternative medication, vasopressin, might be beneficial in neonatal CPR due

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SOX17 Deficiency Mediates Pulmonary Hypertension: At the Crossroads of Sex, Metabolism, and Genetics

Shreya Sangam, Xutong Sun, Tae-Hwi Schwantes-An, Manivannan Yegambaram, Qing Lu, Yinan Shi, Todd Cook, Amanda Fisher, Andrea L. Frump, Anna Coleman, Yanan Sun, Shuxin Liang, Howard Crawford, Katie A. Lutz, Avinash D. Maun, Michael W. Pauciulo, Jason H. Karnes, Ketul R. Chaudhary, Duncan J. Stewart, Paul R. Langlais, Mohit Jain, Mona Alotaibi, Tim Lahm, Yan

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Single-Cell Analysis Reveals Distinct Immune and Smooth Muscle Cell Populations that Contribute to Chronic Thromboembolic Pulmonary Hypertension

Gayathri Viswananthan, Helene Fradin Kirshner, Nour Nazo, Saba Ali, Asvin Ganapathi, Ian Cumming, Youghua Zhuang, Issac Choi, Anmol Warman, Chanpreet Jassal, Susana Almeida-Peters, John Haney, David Corcoran, Yen-Rei Yu, Sudarshan RajagopalUniversity of Colorado Anschutz Medical Campus. Duke University School of Medicine.United States American Journal of Respiratory and Critical Care MedicineAm J Respir Crit Care Med

Single-Cell Analysis Reveals Distinct Immune and Smooth Muscle Cell Populations that Contribute to Chronic Thromboembolic Pulmonary Hypertension Read More »

Pre-Flight Hypoxemia Challenge Testing in Bronchopulmonary Dysplasia

Jonathan C. Levin, Catherine A. Sheils, Lystra P. HaygenBoston Children’s Hospital and Brigham and Women’s Hospital.United States PediatricsPediatrics 2023; 152DOI: 10.1542/peds.2022-061001 AbstractBackground and objectives: Former premature infants with bronchopulmonary dysplasia (BPD) are at risk for hypoxemia during air travel, but it is unclear until what age. We aimed to determine pass rates for high altitude simulation testing

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A mother and daughter with the p.R443X mutation of mucopolysaccharidosis type II: Genotype and phenotype analysis

Young Bae Sohn, Su Jin Kim, Sung Won Park, Hyung-Doo Park, Chang-Seok Ki, Chi Hwa Kim, Seung Won Huh, Sunghee Yeau, Kyung-Hoon Paik, Dong-Kyu JinSamsung Medical Center. Republic of Korea American Journal of Medical GeneticsAm J Med Genet 2010; 152A: 3129-3132DOI: 10.1002/ajmg.a.33589 AbstractMucopolysaccharidosis type II (Hunter syndrome) is a lysosomal storage disease caused by a deficiency

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