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Articles 91 - 117 of 117
Full-Text Articles in Medicine and Health Sciences
Sirolimus Therapy And Follow-Up In A Patient With Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy, Qiong Chen, Yongxing Chen, Xiaohong Wang, Haihua Yang, Yingxian Zhang, Xiaojing Liu, Yun Yan, Haiyan Wei
Sirolimus Therapy And Follow-Up In A Patient With Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy, Qiong Chen, Yongxing Chen, Xiaohong Wang, Haihua Yang, Yingxian Zhang, Xiaojing Liu, Yun Yan, Haiyan Wei
Manuscripts, Articles, Book Chapters and Other Papers
Congenital hyperinsulinism (CHI) is the most common cause of severe, persistent hypoglycemia in neonates and infants. If the patient does not respond to medical treatment the currently available treatment is subtotal pancreatectomy, but some patients still experience severe hypoglycemia after surgery. Sirolimus, a mammalian target of rapamycin inhibitor has recently been reported to be effective in the treatment of insulinoma and CHI patients. Here we report a patient with CHI who had prolonged hypoglycemia after subtotal pancreatectomy. The patient had a heterozygous mutation in ABCC8 but was unresponsive to an optimal dose of diazoxide (15 mg/ kg/day) and octreotide (30 …
Engaging Patients And Parents To Improve Mental Health Intervention For Youth With Rheumatological Disease., Oluwatunmise A. Fawole, Michelle V. Reed, Julia G. Harris, Aimee Hersh, Martha Rodriguez, Karen Onel, Erica Lawson, Tamar Rubinstein, Kaveh Ardalan, Esi Morgan, Anne Paul, Judy Barlin, R Paola Daly, Mitali Dave, Shannon Malloy, Shari Hume, Suzanne Schrandt, Laura Marrow, Angela Chapson, Donna Napoli, Michael Napoli, Miranda Moyer, Vincent Delgaizo, Ashley Danguecan, Emily Von Scheven, Andrea Knight, Carra Investigators
Engaging Patients And Parents To Improve Mental Health Intervention For Youth With Rheumatological Disease., Oluwatunmise A. Fawole, Michelle V. Reed, Julia G. Harris, Aimee Hersh, Martha Rodriguez, Karen Onel, Erica Lawson, Tamar Rubinstein, Kaveh Ardalan, Esi Morgan, Anne Paul, Judy Barlin, R Paola Daly, Mitali Dave, Shannon Malloy, Shari Hume, Suzanne Schrandt, Laura Marrow, Angela Chapson, Donna Napoli, Michael Napoli, Miranda Moyer, Vincent Delgaizo, Ashley Danguecan, Emily Von Scheven, Andrea Knight, Carra Investigators
Manuscripts, Articles, Book Chapters and Other Papers
BACKGROUND: Mental health disorders are common in youth with rheumatological disease yet optimal intervention strategies are understudied in this population. We examined patient and parent perspectives on mental health intervention for youth with rheumatological disease.
METHODS: We conducted a mixed methods cross-sectional study, via anonymous online survey, developed by researchers together with patient/parent partners, to quantitatively and qualitatively examine youth experiences with mental health services and resources in North America. Patients ages 14-24 years with juvenile idiopathic arthritis, juvenile dermatomyositis, or systemic lupus erythematous, and parents of patients ages 8-24 with these diseases were eligible (not required to participate in …
Integrated Multidisciplinary Treatment For Pediatric Inflammatory Bowel Disease., Anava A. Wren, Michele H. Maddux
Integrated Multidisciplinary Treatment For Pediatric Inflammatory Bowel Disease., Anava A. Wren, Michele H. Maddux
Manuscripts, Articles, Book Chapters and Other Papers
No abstract provided.
Racial Disparities In Necrotizing Enterocolitis., Alain Cuna, Venkatesh Sampath, Minesh Khashu
Racial Disparities In Necrotizing Enterocolitis., Alain Cuna, Venkatesh Sampath, Minesh Khashu
Manuscripts, Articles, Book Chapters and Other Papers
Necrotizing enterocolitis (NEC) is a serious disease of the intestinal tract affecting 5-10% of pre-term infants with up to 50% mortality in those that require surgery. There is wide variation in the rates and outcomes of NEC by race and ethnicity, and the reasons for this disparity are poorly understood. In this article, we review the epidemiology and discuss possible explanations for racial and ethnic differences in NEC. Most of the current evidence investigating the role of race in NEC comes from North America and suggests that Hispanic ethnicity and non-Hispanic Black race are associated with higher risk of NEC …
Impaired Eif5a Function Causes A Mendelian Disorder That Is Partially Rescued In Model Systems By Spermidine., Víctor Faundes, Martin D. Jennings, Siobhan Crilly, Sarah Legraie, Sarah E. Withers, Sara Cuvertino, Sally J. Davies, Andrew G L Douglas, Andrew E. Fry, Victoria Harrison, Jeanne Amiel, Daphné Lehalle, William G. Newman, Patricia Newkirk, Judith Ranells, Miranda Splitt, Laura A. Cross, Carol J. Saunders, Bonnie Sullivan, Jorge L. Granadillo, Christopher T. Gordon, Paul R. Kasher, Graham D. Pavitt, Siddharth Banka
Impaired Eif5a Function Causes A Mendelian Disorder That Is Partially Rescued In Model Systems By Spermidine., Víctor Faundes, Martin D. Jennings, Siobhan Crilly, Sarah Legraie, Sarah E. Withers, Sara Cuvertino, Sally J. Davies, Andrew G L Douglas, Andrew E. Fry, Victoria Harrison, Jeanne Amiel, Daphné Lehalle, William G. Newman, Patricia Newkirk, Judith Ranells, Miranda Splitt, Laura A. Cross, Carol J. Saunders, Bonnie Sullivan, Jorge L. Granadillo, Christopher T. Gordon, Paul R. Kasher, Graham D. Pavitt, Siddharth Banka
Manuscripts, Articles, Book Chapters and Other Papers
The structure of proline prevents it from adopting an optimal position for rapid protein synthesis. Poly-proline-tract (PPT) associated ribosomal stalling is resolved by highly conserved eIF5A, the only protein to contain the amino acid hypusine. We show that de novo heterozygous EIF5A variants cause a disorder characterized by variable combinations of developmental delay, microcephaly, micrognathia and dysmorphism. Yeast growth assays, polysome profiling, total/hypusinated eIF5A levels and PPT-reporters studies reveal that the variants impair eIF5A function, reduce eIF5A-ribosome interactions and impair the synthesis of PPT-containing proteins. Supplementation with 1 mM spermidine partially corrects the yeast growth defects, improves the polysome profiles …
Intestinal Stem Cell Development In The Neonatal Gut: Pathways Regulating Development And Relevance To Necrotizing Enterocolitis., Aparna Venkatraman, Wei Yu, Christopher R. Nitkin, Venkatesh Sampath
Intestinal Stem Cell Development In The Neonatal Gut: Pathways Regulating Development And Relevance To Necrotizing Enterocolitis., Aparna Venkatraman, Wei Yu, Christopher R. Nitkin, Venkatesh Sampath
Manuscripts, Articles, Book Chapters and Other Papers
The intestine is extremely dynamic and the epithelial cells that line the intestine get replaced every 3-5 days by highly proliferative intestinal stem cells (ISCs). The instructions for ISCs to self-renew or to differentiate come as cues from their surrounding microenvironment or their niche. A small number of evolutionarily conserved signaling pathways act as a critical regulator of the stem cells in the adult intestine, and these pathways are well characterized. However, the mechanisms, nutritional, and environmental signals that help establish the stem cell niche in the neonatal intestine are less studied. Deciphering the key signaling pathways that regulate the …
Unilateral Nephrectomy Stimulates Erk And Is Associated With Enhanced Na Transport., Robert Repetti, Nomrota Majumder, Karin Carneiro De Oliveira, Jennifer Meth, Tenzin Yangchen, Mukut Sharma, Tarak Srivastava, Rajeev Rohatgi
Unilateral Nephrectomy Stimulates Erk And Is Associated With Enhanced Na Transport., Robert Repetti, Nomrota Majumder, Karin Carneiro De Oliveira, Jennifer Meth, Tenzin Yangchen, Mukut Sharma, Tarak Srivastava, Rajeev Rohatgi
Manuscripts, Articles, Book Chapters and Other Papers
Nephron loss initiates compensatory hemodynamic and cellular effects on the remaining nephrons. Increases in single nephron glomerular filtration rate and tubular flow rate exert higher fluid shear stress (FSS) on tubules. In principal cell (PC) culture models FSS induces ERK, and ERK is implicated in the regulation of transepithelial sodium (Na) transport, as well as, proliferation. Thus, we hypothesize that high tubular flow and FSS mediate ERK activation in the cortical collecting duct (CCD) of solitary kidney which regulates amiloride sensitive Na transport and affects CCD cell number. Immunoblotting of whole kidney protein lysate was performed to determine phospho-ERK (pERK) …
Latent Variables Quantifying Neighborhood Characteristics And Their Associations With Poor Mental Health., Katherine L. Forthman, Janna M. Colaizzi, Hung-Wen Yeh, Rayus Kuplicki, Martin P. Paulus
Latent Variables Quantifying Neighborhood Characteristics And Their Associations With Poor Mental Health., Katherine L. Forthman, Janna M. Colaizzi, Hung-Wen Yeh, Rayus Kuplicki, Martin P. Paulus
Manuscripts, Articles, Book Chapters and Other Papers
Neighborhood characteristics can have profound impacts on resident mental health, but the wide variability in methodologies used across studies makes it difficult to reach a consensus as to the implications of these impacts. The aim of this study was to simplify the assessment of neighborhood influence on mental health. We used a factor analysis approach to reduce the multi-dimensional assessment of a neighborhood using census tracts and demographic data available from the American Community Survey (ACS). Multivariate quantitative characterization of the neighborhood was derived by performing a factor analysis on the 2011-2015 ACS data. The utility of the latent variables …
Reproductive Intentions And Corresponding Use Of Safer Conception Methods And Contraception Among Ugandan Hiv Clients In Serodiscordant Relationships., Glenn J. Wagner, Deborah Mindry, Emily A. Hurley, Jolly Beyeza-Kashesya, Violet Gwokyalya, Sarah Finocchario-Kessler, Rhoda K. Wanyenze, Mastula Nanfuka, Mahlet G. Tebeka, Kathy Goggin
Reproductive Intentions And Corresponding Use Of Safer Conception Methods And Contraception Among Ugandan Hiv Clients In Serodiscordant Relationships., Glenn J. Wagner, Deborah Mindry, Emily A. Hurley, Jolly Beyeza-Kashesya, Violet Gwokyalya, Sarah Finocchario-Kessler, Rhoda K. Wanyenze, Mastula Nanfuka, Mahlet G. Tebeka, Kathy Goggin
Manuscripts, Articles, Book Chapters and Other Papers
CONTEXT: Among people living with HIV in Uganda, desires to have a child and unplanned pregnancies are both common, while utilization of safer conception methods (SCM) and modern contraceptives are low.
METHODS: Three hundred eighty-nine HIV clients who reported considering childbearing with their uninfected partner enrolled in a safer conception counseling intervention trial in Uganda. Multiple regression analysis and baseline data were used to examine correlates of reproductive intentions and behaviors, including use of safer conception methods and contraception.
RESULTS: Most (n = 313; 80.5%) reported that both they and their partner wanted to have a child now, which was …
Combined Use Of The Ketogenic Diet And Vagus Nerve Stimulation In Pediatric Drug-Resistant Epilepsy., Ayman Abdelmoity, Jean-Baptist Lepichon, Sherouk A. Abdelmoity, Ashley K. Sherman, Ara Hall, Ahmed Abdelmoity
Combined Use Of The Ketogenic Diet And Vagus Nerve Stimulation In Pediatric Drug-Resistant Epilepsy., Ayman Abdelmoity, Jean-Baptist Lepichon, Sherouk A. Abdelmoity, Ashley K. Sherman, Ara Hall, Ahmed Abdelmoity
Manuscripts, Articles, Book Chapters and Other Papers
Objective: Patients with drug-resistant epilepsy (DRE) pose considerable management challenges for patients, their families, and providers. Both the vagus nerve stimulator (VNS) and the ketogenic diet (KD) have been shown to be safe and effective in treating DRE. Nevertheless, information is lacking regarding treatment with combination of both modalities. This study reports the efficacy and tolerability of combining VNS and KD in a pediatric cohort with intractable epilepsy.
Methods: This is a retrospective review of 33 patients (0-17 years) with DRE treated with VNS and KD at a single pediatric level IV epilepsy center. We compared seizure reduction rates for …
Functional Constipation Induces Bladder Overactivity Associated With Upregulations Of Htr2 And Trpv2 Pathways., Nao Iguchi, Alonso Carrasco, Alison X. Xie, Ricardo H. Pineda, Anna P. Malykhina, Duncan T. Wilcox
Functional Constipation Induces Bladder Overactivity Associated With Upregulations Of Htr2 And Trpv2 Pathways., Nao Iguchi, Alonso Carrasco, Alison X. Xie, Ricardo H. Pineda, Anna P. Malykhina, Duncan T. Wilcox
Manuscripts, Articles, Book Chapters and Other Papers
Bladder and bowel dysfunction (BBD) is a common yet underdiagnosed paediatric entity that describes lower urinary tract symptoms (LUTS) accompanied by abnormal bowel patterns manifested as constipation and/or encopresis. LUTS usually manifest as urgency, urinary frequency, incontinence, and urinary tract infections (UTI). Despite increasing recognition of BBD as a risk factor for long-term urinary tract problems including recurrent UTI, vesicoureteral reflux, and renal scarring, the mechanisms underlying BBD have been unclear, and treatment remains empirical. We investigated how constipation affects the lower urinary tract function using a juvenile murine model of functional constipation. Following four days of functional constipation, animals …
Utilization Of The Naranjo Scale To Evaluate Adverse Drug Reactions At A Free-Standing Children's Hospital., Madhavi Murali, Sarah Suppes, Keith Feldman, Jennifer Goldman
Utilization Of The Naranjo Scale To Evaluate Adverse Drug Reactions At A Free-Standing Children's Hospital., Madhavi Murali, Sarah Suppes, Keith Feldman, Jennifer Goldman
Manuscripts, Articles, Book Chapters and Other Papers
The relationship between the Naranjo scaling system and pediatric adverse drug reactions (ADR) is poorly understood. We performed a retrospective review of 1,676 pediatric ADRs documented at our hospital from 2014-2018. We evaluated patient demographics, implicated medication, ADR severity, calculated Naranjo score, associated symptoms, and location within the hospital in which the ADR was documented. ADR severity was poorly correlated with Naranjo interpretation. Out of the 10 Naranjo scale questions, 4 had a response of "unknown" greater than 85% of the time. Cardiovascular and oncological/immunologic agents were more likely to have a probable or definite Naranjo interpretation compared to antimicrobials. …
Colostomy Takedown: Ischemic Complication Following Anorectal Malformation Surgery., Wendy Jo Svetanoff, Justin A. Sobrino Md, Grace S. Mitchell, Rebecca M. Rentea
Colostomy Takedown: Ischemic Complication Following Anorectal Malformation Surgery., Wendy Jo Svetanoff, Justin A. Sobrino Md, Grace S. Mitchell, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Introduction: Anorectal malformations (ARM) are complex disorders that often require staged reconstructions. We present a case and imaging findings of a child who developed issues following colostomy closure due to segmental colonic ischemia. Case Presentation. A 3-year-old female with Currarino syndrome presented with abdominal distention, blood-flecked stools, and prolonged cecostomy flush time. For her anorectal malformation, a colostomy was initially placed. A new colostomy was created at posterior sagittal anorectoplasty (PSARP) to allow the distal rectum to reach the anus without tension. Differentials for her presenting symptoms included a mislocation of the anus, stenosis at the anoplasty site, stricture …
Frontiers In Human Factors: Embedding Specialists In Multi-Disciplinary Efforts To Improve Healthcare., Ken Catchpole, Paul Bowie, Sarah Fouquet, Joy Rivera, Sue Hignett
Frontiers In Human Factors: Embedding Specialists In Multi-Disciplinary Efforts To Improve Healthcare., Ken Catchpole, Paul Bowie, Sarah Fouquet, Joy Rivera, Sue Hignett
Manuscripts, Articles, Book Chapters and Other Papers
Despite the application of a huge range of human factors (HF) principles in a growing range of care contexts, there is much more that could be done to realize this expertise for patient benefit, staff well-being and organizational performance. Healthcare has struggled to embrace system safety approaches, misapplied or misinterpreted others, and has stuck to a range of outdated and potentially counter-productive myths even has safety science has developed. One consequence of these persistent misunderstandings is that few opportunities exist in clinical settings for qualified HF professionals. Instead, HF has been applied by clinicians and others, to highly variable degrees-sometimes …
Shear Stress Associated With Cardiopulmonary Bypass Induces Expression Of Inflammatory Cytokines And Necroptosis In Monocytes., Lan N. Tu, Lance Hsieh, Masaki Kajimoto, Kevin Charette, Nataliya Kibiryeva, Adriana Forero, Sarah Hampson, Jennifer A. Marshall, James O'Brien, Marta Scatena, Michael A. Portman, Ram Savan, Chris Benner, Alberto Aliseda, Muhammad Nuri, Douglas Bittel, Peter Pastuzsko, Vishal Nigam
Shear Stress Associated With Cardiopulmonary Bypass Induces Expression Of Inflammatory Cytokines And Necroptosis In Monocytes., Lan N. Tu, Lance Hsieh, Masaki Kajimoto, Kevin Charette, Nataliya Kibiryeva, Adriana Forero, Sarah Hampson, Jennifer A. Marshall, James O'Brien, Marta Scatena, Michael A. Portman, Ram Savan, Chris Benner, Alberto Aliseda, Muhammad Nuri, Douglas Bittel, Peter Pastuzsko, Vishal Nigam
Manuscripts, Articles, Book Chapters and Other Papers
Cardiopulmonary bypass (CPB) is required during most cardiac surgeries. CBP drives systemic inflammation and multiorgan dysfunction that is especially severe in neonatal patients. Limited understanding of molecular mechanisms underlying CPB-associated inflammation presents a significant barrier to improve clinical outcomes. To better understand these clinical issues, we performed mRNA sequencing on total circulating leukocytes from neonatal patients undergoing CPB. Our data identify myeloid cells, particularly monocytes, as the major cell type driving transcriptional responses to CPB. Furthermore, IL-8 and TNF-α were inflammatory cytokines robustly upregulated in leukocytes from both patients and piglets exposed to CPB. To delineate the molecular mechanism, we …
A Promising Approach In Home Visiting To Support Families Affected By Maternal Substance Use., Donna M. O'Malley, Danielle F. Chiang, Emily Siedlik, Katharine Ragon, Marcia Dutcher, Oneta Templeton
A Promising Approach In Home Visiting To Support Families Affected By Maternal Substance Use., Donna M. O'Malley, Danielle F. Chiang, Emily Siedlik, Katharine Ragon, Marcia Dutcher, Oneta Templeton
Manuscripts, Articles, Book Chapters and Other Papers
INTRODUCTION: Many factors influence women's use of alcohol and other drugs while pregnant and postpartum. Substance use impacts the maternal-child relationship during the critical neonatal period. The first days and months of human development lay the foundation for health and well-being across the lifespan, making this period an important window of opportunity to interrupt the transmission of trauma and stress to the next generation. Pregnant and postpartum women with a history of substance use require specialized support services.
METHODS: The Team for Infants Exposed to Substance abuse (TIES) Program provides a holistic, multi-disciplinary, community-based model to address the complex needs …
Melanotic Neuroectodermal Tumor Of Infancy, Mohammad H. Almomani, Rebecca M. Rentea
Melanotic Neuroectodermal Tumor Of Infancy, Mohammad H. Almomani, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Melanotic neuroectodermal tumor of infancy (MNTI) is a rapidly growing benign tumor that arises from the neural crest. It typically occurs in infants in the head and neck region. It was described for the first time in 1918 by Krompecher, and since the origin was unknown, it was described as congenital melanocarcinoma. Until 1966, this tumor was characterized by variable names (e.g., retinal anlage tumor, pigmented congenital epulis, melanotic progonoma, pigmented teratoma, atypical melanoblastoma, etc.). However, Borello and Gorlin found that the tumor produces vanillylmandelic acid (VMA), which is produced by other types of tumors that arise from the neural …
Posterior Urethral Valve, George Bingham, Rebecca M. Rentea
Posterior Urethral Valve, George Bingham, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Posterior urethral valves are one of the most common causes of urinary tract obstruction in the pediatric population. They are obstructing membranous folds in the lumen of the posterior part of the urethra and are exclusive to male patients. Posterior urethral valves were first described by Morgagni in 1717 and then by Langenbeck in 1802, who reported valve-like folds in dissected cadavers. Posterior urethral valves can lead to a spectrum of pathology both inside and outside the urinary system. This includes acute retention, chronic kidney disease, and in severe cases, pulmonary hypoplasia secondary to low amniotic fluid levels.
Posterior urethral …
Thymectomy, Bryan Bennett, Rebecca M. Rentea
Thymectomy, Bryan Bennett, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Excerpt
Thymectomy is the resection of the thymus gland. This anterior mediastinal organ can enlarge as in myasthenia gravis and thymoma and harbor malignant cells such as in thymic carcinoma or neuroendocrine tumors. The first thymectomies were performed incidentally in conjunction with thyroidectomies for Grave disease by Garre and Sauerbruch, but it was not until Blalock and colleagues that multiple series were performed with adequate results.
This also included patients with myasthenia gravis but without thymomas. The gold standard approach for thymectomy is a median sternotomy or transsternal approach, but this has evolved to less invasive techniques such as upper …
Temporary Abdominal Closure Techniques, Laura R. Brown, Rebecca M. Rentea
Temporary Abdominal Closure Techniques, Laura R. Brown, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Critically ill patients with underlying intraabdominal hypertension due to sepsis or injury are often managed with a damage control laparotomy (DCL), which involves the minimum intervention necessary to save the patient’s life. In order to save time, the abdomen may be left open to facilitate a second look to confirm the control of bleeding or sepsis before fascial closure. In the interim, some patients will develop bowel and abdominal wall edema with a high-volume resuscitation, especially if the crystalloid fluid is used primarily.
The many causes and management of intraabdominal hypertension as well as the indications for a damage control …
Tisagenlecleucel Infusion In Patients With Relapsed/Refractory All And Concurrent Serious Infection., Erin Hall, Dwight E. Yin, Rakesh K. Goyal, Atif Ahmed, Grace S. Mitchell, Shawn D. St Peter, Terrie Flatt, Ibrahim A. Ahmed, Weijie Li, Richard J. Hendrickson, Keith August, Douglas Myers
Tisagenlecleucel Infusion In Patients With Relapsed/Refractory All And Concurrent Serious Infection., Erin Hall, Dwight E. Yin, Rakesh K. Goyal, Atif Ahmed, Grace S. Mitchell, Shawn D. St Peter, Terrie Flatt, Ibrahim A. Ahmed, Weijie Li, Richard J. Hendrickson, Keith August, Douglas Myers
Manuscripts, Articles, Book Chapters and Other Papers
BACKGROUND: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T (CAR-T) cell therapy, has demonstrated durable efficacy and a manageable safety profile in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) in the ELIANA pivotal trial and real-world experience. Experience from investigator-led studies prior to ELIANA suggests that infections and inflammatory conditions may exacerbate the severity of cytokine release syndrome (CRS) associated with CAR-T cell therapy, leading to extreme caution and strong restrictions for on-study and commercial infusion of tisagenlecleucel in patients with active infection. CRS intervention with interleukin (IL)-6 blockade and/or steroid therapy was introduced late in …
Study Design And Baseline Characteristics Of The Cardinal Trial: A Phase 3 Study Of Bardoxolone Methyl In Patients With Alport Syndrome., Glenn M. Chertow, Gerald B. Appel, Sharon Andreoli, Sripal Bangalore, Geoffrey A. Block, Arlene B. Chapman, Melanie P. Chin, Keisha L. Gibson, Angie Goldsberry, Kazumoto Iijima, Lesley A. Inker, Bertrand Knebelmann, Laura H. Mariani, Colin J. Meyer, Kandai Nozu, Megan O'Grady, Arnold L. Silva, Peter Stenvinkel, Roser Torra, Bradley A. Warady, Pablo E. Pergola
Study Design And Baseline Characteristics Of The Cardinal Trial: A Phase 3 Study Of Bardoxolone Methyl In Patients With Alport Syndrome., Glenn M. Chertow, Gerald B. Appel, Sharon Andreoli, Sripal Bangalore, Geoffrey A. Block, Arlene B. Chapman, Melanie P. Chin, Keisha L. Gibson, Angie Goldsberry, Kazumoto Iijima, Lesley A. Inker, Bertrand Knebelmann, Laura H. Mariani, Colin J. Meyer, Kandai Nozu, Megan O'Grady, Arnold L. Silva, Peter Stenvinkel, Roser Torra, Bradley A. Warady, Pablo E. Pergola
Manuscripts, Articles, Book Chapters and Other Papers
INTRODUCTION: Alport syndrome is a rare genetic disorder that affects as many as 60,000 persons in the USA and a total of 103,000 persons (10,000) in the European Union [1, 2]. It is the second most common inherited cause of kidney failure and is characterized by progressive loss of kidney function that often leads to end-stage kidney disease. Currently, there are no approved disease-specific agents for therapeutic use. We designed a phase 3 study (CARDINAL; NCT03019185) to evaluate the safety, tolerability, and efficacy of bardoxolone methyl in patients with Alport syndrome.
METHODS: The CARDINAL phase 3 study is an international, …
Recent Advances Of Integrative Bio-Omics Technologies To Improve Type 1 Diabetes (T1d) Care, Nisha Karwal, Megan Rodrigues, David D. Williams, Ryan Mcdonough, Diana Ferro
Recent Advances Of Integrative Bio-Omics Technologies To Improve Type 1 Diabetes (T1d) Care, Nisha Karwal, Megan Rodrigues, David D. Williams, Ryan Mcdonough, Diana Ferro
Manuscripts, Articles, Book Chapters and Other Papers
Type 1 diabetes (T1D) is a complex autoimmune disease that currently cannot be cured, only managed. Optimal treatment the of T1D symptoms, requires a multidisciplinary care team, including endocrinologists, educators, primary care providers, health care specialists, genetic counselors, and data scientists. This review summarizes how an integrative approach to T1D drives innovation and quality improvements in health care. Specifically, we highlight how “-omics” technologies facilitate the understanding of different aspects of the disease, including prevention, pathogenesis, diagnostics, and treatment. Furthermore, we explore how biological data can be combined with personal and electronic health records to tailor medical interventions to the …
Opportunities, Barriers, And Recommendations In Down Syndrome Research., James A. Hendrix, Angelika Amon, Leonard Abbeduto, Stamatis Agiovlasitis, Tarek Alsaied, Heather A. Anderson, Lisa J. Bain, Nicole Baumer, Anita Bhattacharyya, Dusan Bogunovic, Kelly N. Botteron, George Capone, Priya Chandan, Isabelle Chase, Brian Chicoine, Cécile Cieuta-Walti, Lara R. Deruisseau, Sophie Durand, Anna Esbensen, Juan Fortea, Sandra Giménez, Ann-Charlotte Granholm, Laura J. Hahn, Elizabeth Head, Hampus Hillerstrom, Lisa M. Jacola, Matthew P. Janicki, Joan M. Jasien, Angela R. Kamer, Raymond D. Kent, Bernard Khor, Jeanne B. Lawrence, Catherine Lemonnier, Amy Feldman Lewanda, William Mobley, Paul E. Moore, Linda Pollak Nelson, Nicolas M. Oreskovic, Ricardo S. Osorio, David Patterson, Sonja A. Rasmussen, Roger H. Reeves, Nancy Roizen, Stephanie Santoro, Stephanie L. Sherman, Nasreen Talib, Ignacio E. Tapia, Kyle M. Walsh, Steven F. Warren, A Nicole White, Guang William Wong, John S. Yi
Opportunities, Barriers, And Recommendations In Down Syndrome Research., James A. Hendrix, Angelika Amon, Leonard Abbeduto, Stamatis Agiovlasitis, Tarek Alsaied, Heather A. Anderson, Lisa J. Bain, Nicole Baumer, Anita Bhattacharyya, Dusan Bogunovic, Kelly N. Botteron, George Capone, Priya Chandan, Isabelle Chase, Brian Chicoine, Cécile Cieuta-Walti, Lara R. Deruisseau, Sophie Durand, Anna Esbensen, Juan Fortea, Sandra Giménez, Ann-Charlotte Granholm, Laura J. Hahn, Elizabeth Head, Hampus Hillerstrom, Lisa M. Jacola, Matthew P. Janicki, Joan M. Jasien, Angela R. Kamer, Raymond D. Kent, Bernard Khor, Jeanne B. Lawrence, Catherine Lemonnier, Amy Feldman Lewanda, William Mobley, Paul E. Moore, Linda Pollak Nelson, Nicolas M. Oreskovic, Ricardo S. Osorio, David Patterson, Sonja A. Rasmussen, Roger H. Reeves, Nancy Roizen, Stephanie Santoro, Stephanie L. Sherman, Nasreen Talib, Ignacio E. Tapia, Kyle M. Walsh, Steven F. Warren, A Nicole White, Guang William Wong, John S. Yi
Manuscripts, Articles, Book Chapters and Other Papers
BACKGROUND: Recent advances in medical care have increased life expectancy and improved the quality of life for people with Down syndrome (DS). These advances are the result of both pre-clinical and clinical research but much about DS is still poorly understood. In 2020, the NIH announced their plan to update their DS research plan and requested input from the scientific and advocacy community.
OBJECTIVE: The National Down Syndrome Society (NDSS) and the LuMind IDSC Foundation worked together with scientific and medical experts to develop recommendations for the NIH research plan.
METHODS: NDSS and LuMind IDSC assembled over 50 experts across …
Simulation Training And Skill Assessment In Obstetrics And Gynecology, Thomas G. Weiss, Rebecca M. Rentea
Simulation Training And Skill Assessment In Obstetrics And Gynecology, Thomas G. Weiss, Rebecca M. Rentea
Manuscripts, Articles, Book Chapters and Other Papers
Simulation is a valuable teaching tool that increases skill and knowledge acquisition in obstetric and gynecologic clinical scenarios. There are increasing options for simulation from animal and human cadaver, low-fidelity (inanimate simulator), and virtual reality simulation. The role of obstetric and gynecologic simulation also plays a key role in both outcomes-based as well as competency-based medical education.
Simulators were initially created for flight training for pilots in the 1920s, and the ability to reproduce clinical situations has been utilized in obstetric and gynecologic education. Simulations used in medical training were used in the 1960s with standardized patients and mannequins. Simulation …
Thyrotoxic Periodic Paralysis In An Adolescent Male: A Case Report And Literature Review., Luke He, Veronica Lawrence, Wayne V. Moore, Yun Yan
Thyrotoxic Periodic Paralysis In An Adolescent Male: A Case Report And Literature Review., Luke He, Veronica Lawrence, Wayne V. Moore, Yun Yan
Manuscripts, Articles, Book Chapters and Other Papers
Thyrotoxic periodic paralysis (TPP) is rarely seen in children and adolescents. Clinical manifestations in children and adolescents may vary. It is important for clinicians to be aware of this rare and life-threatening condition.
Retrospective Review Of Pharmacogenetic Testing At An Academic Children's Hospital., Timothy A. Roberts, Jennifer A. Wagner, Tracy L. Sandritter, Benjamin T. Black, Andrea Gaedigk, Stephani L. Stancil
Retrospective Review Of Pharmacogenetic Testing At An Academic Children's Hospital., Timothy A. Roberts, Jennifer A. Wagner, Tracy L. Sandritter, Benjamin T. Black, Andrea Gaedigk, Stephani L. Stancil
Manuscripts, Articles, Book Chapters and Other Papers
There is limited evidence to support pharmacogenetic (PGx) testing in children. We conducted a retrospective review of PGx testing among 452 patients at an academic children's hospital to determine the potential utility of PGx in diseases of childhood and to identify targets for future pediatric pharmacogenetic research. An actionable gene-drug pair associated with the 28 genes tested (Clinical Pharmacogenetics Implementation Consortium (CPIC) level A or B, Pharmacogenomics Knowledge Base (PharmGKB) level 1A or B, or US Food and Drug Administration (FDA) recommendation and a PharmGKB level) was present in 98.7% of patients. We identified 203 actionable gene-drug-diagnosis groups based on …