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Articles 6181 - 6210 of 10526
Full-Text Articles in Medicine and Health Sciences
Neurological Complications And Recovery Rates Of Patients With Adult Cervical Deformity Surgeries, Han Jo Kim, Yu-Cheng Yao, Christopher I. Shaffrey, Justin S. Smith, Michael P. Kelly, Munish Gupta, Todd J. Albert, Themistocles S. Protopsaltis, Gregory M. Mundis, Peter Passias, Eric Klineberg, Shay Bess, Virginie Lafage, Christopher P. Ames, International Spine Study Group
Neurological Complications And Recovery Rates Of Patients With Adult Cervical Deformity Surgeries, Han Jo Kim, Yu-Cheng Yao, Christopher I. Shaffrey, Justin S. Smith, Michael P. Kelly, Munish Gupta, Todd J. Albert, Themistocles S. Protopsaltis, Gregory M. Mundis, Peter Passias, Eric Klineberg, Shay Bess, Virginie Lafage, Christopher P. Ames, International Spine Study Group
2020-Current year OA Pubs
STUDY DESIGN: Retrospective cohort study.
OBJECTIVE: This study aims to report the incidence, risk factors, and recovery rate of neurological complications (NC) in patients with adult cervical deformity (ACD) who underwent corrective surgery.
METHODS: ACD patients undergoing surgery from 2013 to 2015 were enrolled in a prospective, multicenter database. Patients were separated into 2 groups according to the presence of neurological complications (NC vs no-NC groups). The types, timing, recovery patterns, and interventions for NC were recorded. Patients' demographics, surgical details, radiographic parameters, and health-related quality of life (HRQOL) scores were compared.
RESULTS: 106 patients were prospectively included. Average age …
Outcomes After Intracranial Rescue Stenting For Acute Ischemic Stroke, Christine Tschoe, Joshua Osbun, Et Al.
Outcomes After Intracranial Rescue Stenting For Acute Ischemic Stroke, Christine Tschoe, Joshua Osbun, Et Al.
2020-Current year OA Pubs
BACKGROUND: In cases of failed recanalization despite modern mechanical thrombectomy (MT) techniques, intracranial rescue stenting (RS) may be beneficial. However, outcomes and complications of RS relative to the natural history of ongoing emergent large vessel occlusion are unknown. To evaluate whether RS for ongoing emergent large vessel occlusion after failed MT achieves superior outcomes to the natural history of persistent emergent large vessel occlusion.
METHODS: Patients from the Stroke Thrombectomy and Aneurysm Registry who underwent RS after failed MT from 2014 to 2019 were analyzed. For outcome comparisons, patients were screened for inclusion/exclusion criteria of 3 major randomized, controlled MT …
Addressing Futility: A Practical Approach, Piroska K. Kopar, Adrienne Visani, Kyler Squirrell, Douglas E. Brown
Addressing Futility: A Practical Approach, Piroska K. Kopar, Adrienne Visani, Kyler Squirrell, Douglas E. Brown
2020-Current year OA Pubs
UNLABELLED: Limiting or withdrawing nonbeneficial medical care is considered ethically responsible throughout most of critical care and medical ethics literature. Practically, however, setting limits to treatment is often challenging. We review the literature to identify best practices for using the definition of futility as an anchoring concept to aid the ethical practice of ICU clinicians.
DATA SOURCES: Source data were obtained from a PubMed literature review.
STUDY SELECTION: English language articles were chosen based on relevance to medical futility ethics, end-of-life care in the ICU, or communication and conflict mitigation strategies.
DATA EXTRACTION: Independent evaluation of selected articles for recurrent …
Infection And Inflammation: New Perspectives On Alzheimer's Disease, Heather E Whitson, Robyn S Klein, Et Al.
Infection And Inflammation: New Perspectives On Alzheimer's Disease, Heather E Whitson, Robyn S Klein, Et Al.
2020-Current year OA Pubs
Neuroinflammation has been recognized as a component of Alzheimer's Disease (AD) pathology since the original descriptions by Alois Alzheimer and a role for infections in AD pathogenesis has long been hypothesized. More recently, this hypothesis has gained strength as human genetics and experimental data suggest key roles for inflammatory cells in AD pathogenesis. To review this topic, Duke/University of North Carolina (Duke/UNC) Alzheimer's Disease Research Center hosted a virtual symposium: "Infection and Inflammation: New Perspectives on Alzheimer's Disease (AD)." Participants considered current evidence for and against the hypothesis that AD could be caused or exacerbated by infection or commensal microbes. …
Treatment Patterns And Standardized Outcome Assessments Among Patients With Inflammatory Conditions Of The Pouch In A Prospective Multicenter Registry, Edward L Barnes, Parakkal Deepak, Poonam Beniwal-Patel, Laura Raffals, Maia Kayal, Marla Dubinsky, Shannon Chang, Peter D R Higgins, Jennifer I Barr, Joseph Galanko, Yue Jiang, Raymond K Cross, Millie D Long, Hans H Herfarth
Treatment Patterns And Standardized Outcome Assessments Among Patients With Inflammatory Conditions Of The Pouch In A Prospective Multicenter Registry, Edward L Barnes, Parakkal Deepak, Poonam Beniwal-Patel, Laura Raffals, Maia Kayal, Marla Dubinsky, Shannon Chang, Peter D R Higgins, Jennifer I Barr, Joseph Galanko, Yue Jiang, Raymond K Cross, Millie D Long, Hans H Herfarth
2020-Current year OA Pubs
BACKGROUND: Much of our understanding about the natural history of pouch-related disorders has been generated from selected populations. We designed a geographically diverse, prospective registry to study the disease course among patients with 1 of 4 inflammatory conditions of the pouch. The primary objectives in this study were to demonstrate the feasibility of a prospective pouch registry and to evaluate the predominant treatment patterns for pouch-related disorders.
METHODS: We used standardized diagnostic criteria to prospectively enroll patients with acute pouchitis, chronic antibiotic-dependent pouchitis (CADP), chronic antibiotic refractory pouchitis (CARP), or Crohn's disease (CD) of the pouch. We obtained detailed clinical …
Motor Network Reorganization Induced In Chronic Stroke Patients With The Use Of A Contralesionally-Controlled Brain Computer Interface, Joseph B. Humphries, Daniela J S Mattos, Jerrel Rutlin, Andy G S Daniel, Kathleen Rybczynski, Theresa Notestine, Joshua S. Shimony, Harold Burton, Alexandre Carter, Eric C. Leuthardt
Motor Network Reorganization Induced In Chronic Stroke Patients With The Use Of A Contralesionally-Controlled Brain Computer Interface, Joseph B. Humphries, Daniela J S Mattos, Jerrel Rutlin, Andy G S Daniel, Kathleen Rybczynski, Theresa Notestine, Joshua S. Shimony, Harold Burton, Alexandre Carter, Eric C. Leuthardt
2020-Current year OA Pubs
No abstract provided.
Epigenomic Analysis Reveals Prevalent Contribution Of Transposable Elements To Cis-Regulatory Elements, Tissue-Specific Expression, And Alternative Promoters In Zebrafish, Hyung Joo Lee, Yiran Hou, Ju Heon Maeng, Nakul M Shah, Yujie Chen, Heather A Lawson, Hongbo Yang, Feng Yue, Ting Wang
Epigenomic Analysis Reveals Prevalent Contribution Of Transposable Elements To Cis-Regulatory Elements, Tissue-Specific Expression, And Alternative Promoters In Zebrafish, Hyung Joo Lee, Yiran Hou, Ju Heon Maeng, Nakul M Shah, Yujie Chen, Heather A Lawson, Hongbo Yang, Feng Yue, Ting Wang
2020-Current year OA Pubs
Transposable elements (TEs) encode regulatory elements that impact gene expression in multiple species, yet a comprehensive analysis of zebrafish TEs in the context of gene regulation is lacking. Here, we systematically investigate the epigenomic and transcriptomic landscape of TEs across 11 adult zebrafish tissues using multidimensional sequencing data. We find that TEs contribute substantially to a diverse array of regulatory elements in the zebrafish genome and that 37% of TEs are positioned in active regulatory states in adult zebrafish tissues. We identify TE subfamilies enriched in highly specific regulatory elements among different tissues. We use transcript assembly to discover TE-derived …
Long Covid After Breakthrough Sars-Cov-2 Infection, Ziyad Al-Aly, Benjamin Bowe, Yan Xie
Long Covid After Breakthrough Sars-Cov-2 Infection, Ziyad Al-Aly, Benjamin Bowe, Yan Xie
2020-Current year OA Pubs
The post-acute sequelae of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection-also referred to as Long COVID-have been described, but whether breakthrough SARS-CoV-2 infection (BTI) in vaccinated people results in post-acute sequelae is not clear. In this study, we used the US Department of Veterans Affairs national healthcare databases to build a cohort of 33,940 individuals with BTI and several controls of people without evidence of SARS-CoV-2 infection, including contemporary (n = 4,983,491), historical (n = 5,785,273) and vaccinated (n = 2,566,369) controls. At 6 months after infection, we show that, beyond the first 30 days of illness, compared to …
Mitochondrial Rna Modifications Shape Metabolic Plasticity In Metastasis, Sylvain Delaunay, Gloria Pascual, Bohai Feng, Kevin Klann, Mikaela Behm, Agnes Hotz-Wagenblatt, Karsten Richter, Karim Zaoui, Esther Herpel, Christian Münch, Sabine Dietmann, Jochen Hess, Salvador Aznar Benitah, Michaela Frye
Mitochondrial Rna Modifications Shape Metabolic Plasticity In Metastasis, Sylvain Delaunay, Gloria Pascual, Bohai Feng, Kevin Klann, Mikaela Behm, Agnes Hotz-Wagenblatt, Karsten Richter, Karim Zaoui, Esther Herpel, Christian Münch, Sabine Dietmann, Jochen Hess, Salvador Aznar Benitah, Michaela Frye
2020-Current year OA Pubs
Aggressive and metastatic cancers show enhanced metabolic plasticity
Retrospective Analysis Of Pacritinib In Patients With Myelofibrosis And Severe Thrombocytopenia, Srdan Verstovsek, Ruben Mesa, Moshe Talpaz, Jean-Jacques Kiladjian, Claire N. Harrison, Stephen T. Oh, Alessandro M. Vannucchi, Raajit Rampal, Bart L. Scott, Sarah A. Buckley, Adam R. Craig, Karisse Roman-Torres, John O. Mascarenhas
Retrospective Analysis Of Pacritinib In Patients With Myelofibrosis And Severe Thrombocytopenia, Srdan Verstovsek, Ruben Mesa, Moshe Talpaz, Jean-Jacques Kiladjian, Claire N. Harrison, Stephen T. Oh, Alessandro M. Vannucchi, Raajit Rampal, Bart L. Scott, Sarah A. Buckley, Adam R. Craig, Karisse Roman-Torres, John O. Mascarenhas
2020-Current year OA Pubs
Thrombocytopenia is common in patients with myelofibrosis (MF) and is a well-established adverse prognostic factor. Both of the approved Janus kinase (JAK) inhibitors, ruxolitinib and fedratinib, can worsen thrombocytopenia and have not been evaluated in patients with severe thrombocytopenia (platelet counts <50×109/L). Pacritinib, a novel JAK2/interleukin-1 receptor-associated kinase 1 inhibitor, has been studied in two phase III trials (PERSIST-1 and PERSIST- 2), both of which enrolled patients with MF and severe thrombocytopenia. In order to better characterize treatment outcomes for this population with advanced disease, we present a retrospective analysis of efficacy and safety data in the 189 patients with severe thrombocytopenia treated in the PERSIST studies. The proportion of patients in the pacritinib group meeting efficacy endpoints was greater than in the BAT group for ≥35% spleen volume reduction (23% vs. 2%, P=0.0007), ≥50% modified Total Symptom Score reduction (25% vs. 8%, P=0.044), and self-reported symptom benefit ("much" or "very much" improved; 25% vs. 8%, P=0.016) at the primary analysis time point (week 24). The adverse event profile of pacritinib was manageable, and dose modification was rarely required. There was no excess in bleeding or death in pacritinib-treated patients. These results indicate that pacritinib is a promising treatment for patients with MF who lack safe and effective therapeutic options due to severe thrombocytopenia.
Developing Shelf-Stable Microbiota Directed Complementary Food (Mdcf) Prototypes For Malnourished Children: Study Protocol For A Randomized, Single-Blinded, Clinical Study, Ishita Mostafa, Shah Mohammad Fahim, Subhasish Das, Md Amran Gazi, Md Mehedi Hasan, Kazi Nazmus Saqeeb, Mustafa Mahfuz, Hannah B Lynn, Michael J Barratt, Jeffrey I Gordon, Tahmeed Ahmed
Developing Shelf-Stable Microbiota Directed Complementary Food (Mdcf) Prototypes For Malnourished Children: Study Protocol For A Randomized, Single-Blinded, Clinical Study, Ishita Mostafa, Shah Mohammad Fahim, Subhasish Das, Md Amran Gazi, Md Mehedi Hasan, Kazi Nazmus Saqeeb, Mustafa Mahfuz, Hannah B Lynn, Michael J Barratt, Jeffrey I Gordon, Tahmeed Ahmed
2020-Current year OA Pubs
BACKGROUND: Childhood undernutrition is a major public health concern that needs special attention to achieve 2025 global nutrition targets. Moderate acute malnutrition (MAM), manifest as wasting (low weight-for-height), affects 33 million children under 5, yet there are currently no global guidelines for its treatment. We recently performed a randomized-controlled clinical study of a microbiota-directed complementary food formulation (MDCF-2) in 12-18-month-old Bangladeshi children with MAM. The results revealed that MDCF-2, freshly prepared each day, produced a significantly greater improvement in ponderal growth than a standard ready-to-use supplementary food (RUSF), an effect that is associated with repair of the disrupted gut microbial …
Antibiotics For Preventing Recurrent Urinary Tract Infection: Systematic Review And Meta-Analysis, Philipp Jent, Julia Berger, Annette Kuhn, Barbara W Trautner, Andrew Atkinson, Jonas Marschall
Antibiotics For Preventing Recurrent Urinary Tract Infection: Systematic Review And Meta-Analysis, Philipp Jent, Julia Berger, Annette Kuhn, Barbara W Trautner, Andrew Atkinson, Jonas Marschall
2020-Current year OA Pubs
Recurrent urinary tract infections are a common health problem. The only comprehensive synthesis on antibiotic prophylaxis in the last 15 years has been a guideline-embedded meta-analysis. We conducted a systematic review and meta-analysis of randomized controlled trials published up to October 13, 2020, evaluating patients age ≥12 years with either ≥2 episodes of lower urinary tract infection (UTI) within 6 months or ≥3 in the past year. Placebo or antibiotics were allowed as comparators. Study quality was low. In the 11 placebo-controlled trials, the risk for developing UTI was 85% lower with prophylaxis in comparison with placebo (risk ratio [RR], …
Onasemnogene Abeparvovec For Presymptomatic Infants With Three Copies Of Smn2 At Risk For Spinal Muscular Atrophy: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al
Onasemnogene Abeparvovec For Presymptomatic Infants With Three Copies Of Smn2 At Risk For Spinal Muscular Atrophy: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al
2020-Current year OA Pubs
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated within six postnatal weeks. Of 15 children with three SMN2 copies treated before symptom onset, all stood independently before 24 months (P < 0.0001; 14 within normal developmental window), and 14 walked independently (P < 0.0001; 11 within normal developmental window). All survived without permanent ventilation at 14 months; ten (67%) maintained body weight (≥3rd WHO percentile) without feeding support through 24 months; and none required nutritional or respiratory support. No serious adverse events were considered treatment-related by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for presymptomatic infants at risk of SMA type 2, underscoring the urgency of early identification and intervention.
Prediction Of Patient Outcomes Through Social Determinants Of Health: The Pulmonary Hypertension Association Registry (Phar) Evaluation, Dan Grinnan, Murali Chakinala, Et Al.
Prediction Of Patient Outcomes Through Social Determinants Of Health: The Pulmonary Hypertension Association Registry (Phar) Evaluation, Dan Grinnan, Murali Chakinala, Et Al.
2020-Current year OA Pubs
Outcomes of patients with pulmonary arterial hypertension (PAH) may be associated with social determinants of health (SDOH) and other baseline patient characteristics. At present, there is no prognostic model to predict important patient outcomes in PAH based on SDOH. Utilizing information from the Pulmonary Hypertension Association Registry (PHAR), we derive a model (PHAR Evaluation or PHARE) to predict an important composite patient outcomes based on SDOH and other patient characteristics. Baseline data regarding SDOH from adult patients with PAH enrolled in the PHAR between 2015 and March 23, 2020, were included for analysis. We performed repeated measures logistic regression modeling …
Management Of Histoplasmosis By Infectious Disease Physicians, Patrick B Mazi, Sandra R Arnold, John W Baddley, Nathan C Bahr, Susan E Beekmann, Todd P Mccarty, Philip M Polgreen, Adriana M Rauseo, Andrej Spec
Management Of Histoplasmosis By Infectious Disease Physicians, Patrick B Mazi, Sandra R Arnold, John W Baddley, Nathan C Bahr, Susan E Beekmann, Todd P Mccarty, Philip M Polgreen, Adriana M Rauseo, Andrej Spec
2020-Current year OA Pubs
BACKGROUND: The Infectious Diseases Society of America (IDSA) guidelines for the management of histoplasmosis were last revised 15 years ago. Since those guidelines were compiled, new antifungal treatment options have been developed. Furthermore, the ongoing development of immunomodulatory therapies has increased the population at increased risk to develop histoplasmosis.
METHODS: An electronic survey about the management practices of histoplasmosis was distributed to the adult infectious disease (ID) physician members of the IDSA's Emerging Infections Network.
RESULTS: The survey response rate was 37% (551/1477). Only 46% (253/551) of respondents reported seeing patients with histoplasmosis. Regions considered endemic had 82% (158/193) of …
A Staged Reconstruction Technique Utilizing Bioprosthetic Mesh Reinforcement In The Repair Of Giant Omphalocele, Annahita R. Fotouhi, Justin M. Sacks, Martin S. Keller, Kamlesh B. Patel
A Staged Reconstruction Technique Utilizing Bioprosthetic Mesh Reinforcement In The Repair Of Giant Omphalocele, Annahita R. Fotouhi, Justin M. Sacks, Martin S. Keller, Kamlesh B. Patel
2020-Current year OA Pubs
No abstract provided.
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity Of Onasemnogene Abeparvovec In Cynomolgus Monkeys, Francis Fonyuy Tukov, Keith Mansfield, Mark Milton, Emily Meseck, Kelley Penraat, Deepa Chand, Andreas Hartmann
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity Of Onasemnogene Abeparvovec In Cynomolgus Monkeys, Francis Fonyuy Tukov, Keith Mansfield, Mark Milton, Emily Meseck, Kelley Penraat, Deepa Chand, Andreas Hartmann
2020-Current year OA Pubs
Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children < 2 years. For later-onset patients, intrathecal onasemnogene abeparvovec may be advantageous over intravenous administration. Recently, microscopic dorsal root ganglion (DRG) changes were observed in nonhuman primates (NHPs) following intrathecal onasemnogene abeparvovec administration. To characterize these DRG findings, two NHP studies evaluating intrathecal onasemnogene abeparvovec administration were conducted: a 12-month study with a 6-week interim cohort and a 13-week study with a 2-week interim cohort. The latter investigated the potential impact of prednisolone or rituximab plus everolimus on DRG toxicity. An additional 6-month, single-dose, intravenous NHP study conducted in parallel evaluated onasemnogene abeparvovec safety (including DRG toxicity) with or without prednisolone coadministration. Intrathecal onasemnogene abeparvovec administration was well tolerated and not associated with clinical observations. Microscopic onasemnogene abeparvovec-related changes were observed in the DRG and trigeminal ganglion (TG) and included mononuclear cell inflammation and/or neuronal degeneration, which was colocalized with high vector transcript expression at 6 weeks postdose. Incidence and severity of DRG changes were generally decreased after 52 weeks compared with 6 weeks postdose. Other onasemnogene abeparvovec-related microscopic findings of axonal degeneration, mononuclear cell infiltrates and/or gliosis in the spinal cord, dorsal spinal nerve root/spinal nerves, and/or peripheral nerves were absent or found at decreased incidences and/or severities after 52 weeks. DRG and/or TG microscopic findings following intravenous onasemnogene abeparvovec dosing included minimal to slight neuronal degeneration and mononuclear cell inflammation at 6 weeks and 6 months postdose. Nervous system microscopic findings following intrathecal onasemnogene abeparvovec (≥1.2 × 10
Design Of A Randomized, Placebo-Controlled, Phase 3 Trial Of Tofersen Initiated In Clinically Presymptomatic Sod1 Variant Carriers: The Atlas Study, Michael Benatar, Robert C Bucelli, Et Al.
Design Of A Randomized, Placebo-Controlled, Phase 3 Trial Of Tofersen Initiated In Clinically Presymptomatic Sod1 Variant Carriers: The Atlas Study, Michael Benatar, Robert C Bucelli, Et Al.
2020-Current year OA Pubs
Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes of ALS has made it difficult to target upstream biological mechanisms of disease, and therapeutic interventions are usually administered relatively late in the course of disease. Genetic forms of ALS offer a unique opportunity for therapeutic development, as genetic associations may reveal potential insights into disease etiology. Genetic ALS may also be amenable to investigating earlier intervention given the possibility of identifying clinically presymptomatic, at-risk individuals with causative genetic variants. There is increasing evidence for a presymptomatic phase of …
Association Between The Covid-19 Pandemic And Insurance-Based Disparities In Mortality After Major Surgery Among Us Adults, Laurent G Glance, Andrew W Dick, Ernie Shippey, Patrick J Mccormick, Richard Dutton, Patricia W Stone, Jingjing Shang, Stewart J Lustik, Heather L Lander, Igor Gosev, Karen E Joynt Maddox
Association Between The Covid-19 Pandemic And Insurance-Based Disparities In Mortality After Major Surgery Among Us Adults, Laurent G Glance, Andrew W Dick, Ernie Shippey, Patrick J Mccormick, Richard Dutton, Patricia W Stone, Jingjing Shang, Stewart J Lustik, Heather L Lander, Igor Gosev, Karen E Joynt Maddox
2020-Current year OA Pubs
Importance: The COVID-19 pandemic caused significant disruptions in surgical care. Whether these disruptions disproportionately impacted economically disadvantaged individuals is unknown.
Objective: To evaluate the association between the COVID-19 pandemic and mortality after major surgery among patients with Medicaid insurance or without insurance compared with patients with commercial insurance.
Design, Setting, and Participants: This cross-sectional study used data from the Vizient Clinical Database for patients who underwent major surgery at hospitals in the US between January 1, 2018, and May 31, 2020.
Exposures: The hospital proportion of patients with COVID-19 during the first wave of COVID-19 cases between March 1 and …
Comparative Effectiveness Of Diversion Of Cerebrospinal Fluid For Children With Severe Traumatic Brain Injury, Michael J Bell, Jose Pineda, Et Al.
Comparative Effectiveness Of Diversion Of Cerebrospinal Fluid For Children With Severe Traumatic Brain Injury, Michael J Bell, Jose Pineda, Et Al.
2020-Current year OA Pubs
Importance: Diversion of cerebrospinal fluid (CSF) has been used for decades as a treatment for children with severe traumatic brain injury (TBI) and is recommended by evidenced-based guidelines. However, these recommendations are based on limited studies.
Objective: To determine whether CSF diversion is associated with improved Glasgow Outcome Score-Extended for Pediatrics (GOS-EP) and decreased intracranial pressure (ICP) in children with severe TBI.
Design, Setting, and Participants: This observational comparative effectiveness study was performed at 51 clinical centers that routinely care for children with severe TBI in 8 countries (US, United Kingdom, Spain, the Netherlands, Australia, New Zealand, South Africa, and …
Onasemnogene Abeparvovec For Presymptomatic Infants With Two Copies Of Smn2 At Risk For Spinal Muscular Atrophy Type 1: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al
Onasemnogene Abeparvovec For Presymptomatic Infants With Two Copies Of Smn2 At Risk For Spinal Muscular Atrophy Type 1: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al
2020-Current year OA Pubs
SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SMN2, expected to develop spinal muscular atrophy (SMA) type 1. Efficacy was compared with a matched Pediatric Neuromuscular Clinical Research natural-history cohort (n = 23). All 14 enrolled infants sat independently for ≥30 seconds at any visit ≤18 months (Bayley-III item #26; P < 0.001; 11 within the normal developmental window). All survived without permanent ventilation at 14 months as per protocol; 13 maintained body weight (≥3rd WHO percentile) through 18 months. No child used nutritional or respiratory support. No serious adverse events were considered related to treatment by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for children expected to develop SMA type 1, highlighting the urgency for universal newborn screening.
Unilateral Loss Of Maxillary Molars In Young Mice Leads To Bilateral Condylar Adaptation And Degenerative Disease., Christopher Phillip Chen, Jiehua Zhang, Bin Zhang, Mohamed G Hassan, Kyle Hane, Caroline C Chen, Ana Alejandra Navarro Palacios, Sunil Kapila, Andrew H Jheon, Alice F Goodwin
Unilateral Loss Of Maxillary Molars In Young Mice Leads To Bilateral Condylar Adaptation And Degenerative Disease., Christopher Phillip Chen, Jiehua Zhang, Bin Zhang, Mohamed G Hassan, Kyle Hane, Caroline C Chen, Ana Alejandra Navarro Palacios, Sunil Kapila, Andrew H Jheon, Alice F Goodwin
2020-Current year OA Pubs
The adaptive response of the mandible and temporomandibular joint (TMJ) to altered occlusion in juvenile patients is presently unclear. To address this question, we established a mouse model in which all molars were extracted from the maxillary right quadrant in prepubertal, 3-week-old mice and analyzed morphological, tissue, cellular, and molecular changes in the mandible and condyle 3 weeks later. Unilateral loss of maxillary molars led to significant, robust, bilateral changes, primarily in condylar morphology, including anteroposterior narrowing of the condylar head and neck and increased convexity at the condylar surface, as determined by geometric morphometric analysis. Furthermore, both condyles in …
Caspase-1-Driven Neutrophil Pyroptosis And Its Role In Host Susceptibility To Pseudomonas Aeruginosa, Karin Santoni, Christine T. N. Pham, Et Al
Caspase-1-Driven Neutrophil Pyroptosis And Its Role In Host Susceptibility To Pseudomonas Aeruginosa, Karin Santoni, Christine T. N. Pham, Et Al
2020-Current year OA Pubs
Multiple regulated neutrophil cell death programs contribute to host defense against infections. However, despite expressing all necessary inflammasome components, neutrophils are thought to be generally defective in Caspase-1-dependent pyroptosis. By screening different bacterial species, we found that several Pseudomonas aeruginosa (P. aeruginosa) strains trigger Caspase-1-dependent pyroptosis in human and murine neutrophils. Notably, deletion of Exotoxins U or S in P. aeruginosa enhanced neutrophil death to Caspase-1-dependent pyroptosis, suggesting that these exotoxins interfere with this pathway. Mechanistically, P. aeruginosa Flagellin activates the NLRC4 inflammasome, which supports Caspase-1-driven interleukin (IL)-1β secretion and Gasdermin D (GSDMD)-dependent neutrophil pyroptosis. Furthermore, P. aeruginosa-induced GSDMD activation …
Is Combined Surgical Dislocation And Proximal Femoral Osteotomy A Safe Procedure For Correction Of Complex Hip Deformities?, Frank W Parilla, Jeffrey J Nepple, Gail E Pashos, Perry L Schoenecker, John C Clohisy
Is Combined Surgical Dislocation And Proximal Femoral Osteotomy A Safe Procedure For Correction Of Complex Hip Deformities?, Frank W Parilla, Jeffrey J Nepple, Gail E Pashos, Perry L Schoenecker, John C Clohisy
2020-Current year OA Pubs
Complex deformities of the hip requiring intra-articular and proximal femoral correction are challenging with regard to surgical access and complication risk. Combined surgical dislocation and proximal femoral osteotomy (SD/PFO) is a surgical strategy that provides unrestricted access to the joint with the capability for adjunctive PFO. Although providing excellent surgical access, concerns over a potentially high risk of postoperative complications remain, and published information on the safety of this technique remain scarce. In this study, we defined the early complication profile of combined surgery across 48 hips with a variety of complex deformities using a standardized, validated complication grading scheme …
Changes In Adiposity Over The Life Course And Gene Expression In Postmenopausal Women, Yunan Han, Graham A Colditz, Adetunji T Toriola
Changes In Adiposity Over The Life Course And Gene Expression In Postmenopausal Women, Yunan Han, Graham A Colditz, Adetunji T Toriola
2020-Current year OA Pubs
BACKGROUND: Early life adiposity and changes in adiposity over the life course are associated with mammographic breast density among postmenopausal women. However, the underlying mechanisms are unknown; therefore, we comprehensively examined the associations of early life body mass index (BMI) and changes in BMI from ages 10, 18 to age at mammogram with growth factor, RANK pathway, and sex hormone gene expression in 372 postmenopausal women.
METHODS: We estimated early life BMI at age 10 using the validated 9-level Stunkard pictogram. We calculated BMI at other ages (18, 30, and current age at mammogram) by dividing weight in kilograms at …
Community-Based Trial Assessing The Impact Of Annual Versus Semiannual Mass Drug Administration With Ivermectin Plus Albendazole And Praziquantel On Helminth Infections In Northwestern Liberia, Obiora A Eneanya, Lincoln Gankpala, Charles W Goss, Aaron T Momolu, Enoch S Nyan, Emmanuel B Gray, Kerstin Fischer, Kurt Curtis, Fatorma K Bolay, Gary J Weil, Peter U Fischer
Community-Based Trial Assessing The Impact Of Annual Versus Semiannual Mass Drug Administration With Ivermectin Plus Albendazole And Praziquantel On Helminth Infections In Northwestern Liberia, Obiora A Eneanya, Lincoln Gankpala, Charles W Goss, Aaron T Momolu, Enoch S Nyan, Emmanuel B Gray, Kerstin Fischer, Kurt Curtis, Fatorma K Bolay, Gary J Weil, Peter U Fischer
2020-Current year OA Pubs
We assessed the impact of three annual vs five semiannual rounds of mass drug administration (MDA) with ivermectin plus albendazole followed by praziquantel for the control or elimination of lymphatic filariasis (LF), onchocerciasis, soil-transmitted helminth (STH) infections and schistosomiasis in Lofa County, Liberia. The study started in 2012 and was interrupted in 2014 during the Ebola virus outbreak. Repeated cross-sectional surveys were conducted in individuals 5 years and older to measure infection markers. Wuchereria bancrofti antigenemia prevalences decreased from 12.5 to 1.2% (90% reduction) and from 13.6 to 4.2% (69% reduction) one year after three rounds of annual or five …
Environmental Conditions To Promote Healthy Childhood Brain/Behavioral Development: Informing Early Preventive Interventions For Delivery In Routine Care, Joan L Luby, Cynthia Rogers, Katie A Mclaughlin
Environmental Conditions To Promote Healthy Childhood Brain/Behavioral Development: Informing Early Preventive Interventions For Delivery In Routine Care, Joan L Luby, Cynthia Rogers, Katie A Mclaughlin
2020-Current year OA Pubs
Environmental experiences early in life have strong and enduring consequences for cognitive, emotional, and neurobiological development and related physical and mental health trajectories. The powerful influence of early caregiver nurturance and stimulation on promoting positive neurodevelopmental outcomes has been demonstrated across species. These findings elucidate the environmental conditions known to facilitate healthy neurodevelopment and underscore the potential for modifiable psychosocial factors in the environment to be harnessed to inform early preventive interventions to promote health and adaptive development. A framework for early preventive interventions to enhance nurturing and responsive caregiving for implementation during early sensitive periods of brain development delivered …
A Phase 2 Trial Of A Topical Antiseptic Bundle In Head And Neck Cancer Surgery: Effects On Surgical Site Infection And The Oral Microbiome, Joseph Zenga, Sidharth V Puram, Et Al.
A Phase 2 Trial Of A Topical Antiseptic Bundle In Head And Neck Cancer Surgery: Effects On Surgical Site Infection And The Oral Microbiome, Joseph Zenga, Sidharth V Puram, Et Al.
2020-Current year OA Pubs
BACKGROUND: Head and neck cancer (HNC) surgery remains an important component of management but is associated with a high rate of surgical site infection (SSI). We aimed to assess the safety and efficacy of a topical mucosal antiseptic bundle in preventing SSI and evaluate microbial predictors of infection through a genomic sequencing approach.
METHODS: This study was an open-label, single-arm, single-center, phase 2 trial of a topical mucosal antiseptic bundle in patients with HNC undergoing aerodigestive tract resection and reconstruction. Patients underwent topical preparation of the oral mucosa with povidone-iodine (PI) and chlorhexidine gluconate (CHG) pre- and intra-operatively followed by …
Interim Outcomes Of Mechanical Thrombectomy For Deep Vein Thrombosis From The All-Comer Clout Registry, David J Dexter, Luis Sanchez, Et Al.
Interim Outcomes Of Mechanical Thrombectomy For Deep Vein Thrombosis From The All-Comer Clout Registry, David J Dexter, Luis Sanchez, Et Al.
2020-Current year OA Pubs
OBJECTIVES: The multicenter, prospective, single arm CLOUT registry assesses the safety and effectiveness of the ClotTriever System (Inari Medical, Irvine, CA) for the treatment of acute and nonacute lower extremity deep vein thrombosis (DVT) in all-comer patients. Reported here are the outcomes of the first 250 patients.
METHODS: All-comer patients with lower extremity DVT were enrolled, including those with bilateral DVT, those with previously failed DVT treatment, and regardless of symptom duration. The primary effectiveness end point is complete or near-complete (≥75%) thrombus removal determined by independent core laboratory-adjudicated Marder scores. Safety outcomes include serious adverse events through 30 days …
Longitudinal Morphological And Functional Characterization Of Human Heart Organoids Using Optical Coherence Tomography, Yixuan Ming, Senyue Hao, Fei Wang, Yonatan R. Lewis-Israeli, Brett D. Volmert, Zhiyao Xu, Anna Goestenkors, Aitor Aguirre, Chao Zhou
Longitudinal Morphological And Functional Characterization Of Human Heart Organoids Using Optical Coherence Tomography, Yixuan Ming, Senyue Hao, Fei Wang, Yonatan R. Lewis-Israeli, Brett D. Volmert, Zhiyao Xu, Anna Goestenkors, Aitor Aguirre, Chao Zhou
2020-Current year OA Pubs
Organoids play an increasingly important role as in vitro models for studying organ development, disease mechanisms, and drug discovery. Organoids are self-organizing, organ-like three-dimensional (3D) cell cultures developing organ-specific cell types and functions. Recently, three groups independently developed self-assembling human heart organoids (hHOs) from human pluripotent stem cells (hPSCs). In this study, we utilized a customized spectral-domain optical coherence tomography (SD-OCT) system to characterize the growth of hHOs. Development of chamber structures and beating patterns of the hHOs were observed via OCT and calcium imaging. We demonstrated the capability of OCT to produce 3D images in a fast, label-free, and …