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Articles 691 - 720 of 20184

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Liver Injury In A Preclinical Model Of Short Bowel Syndrome Is Associated With Impaired Autophagy, Daniel M Alligood, Alex Park, Kerry Swanson, Naima Pyarali, Evan Mayse, David M Alvarado, Xiuli Liu, Jae-Sung Kim, Nicholas O Davidson, Deborah C Rubin, Jun Guo, Brad W Warner Dec 2025

Liver Injury In A Preclinical Model Of Short Bowel Syndrome Is Associated With Impaired Autophagy, Daniel M Alligood, Alex Park, Kerry Swanson, Naima Pyarali, Evan Mayse, David M Alvarado, Xiuli Liu, Jae-Sung Kim, Nicholas O Davidson, Deborah C Rubin, Jun Guo, Brad W Warner

2020-Current year OA Pubs

Critical for cellular maintenance and repair, autophagy recycles damaged or redundant cellular components. Dysregulated autophagy is associated with progressive cellular injury in various liver pathologies. However, the role of autophagy in intestinal failure-associated liver disease has not previously been investigated. Using a preclinical murine model of short bowel syndrome, we explored the role of autophagy impairment at early and late time points. Regardless of sex, small bowel resected (SBR) mice demonstrated significant liver injury compared to sham controls, exhibiting elevated serum transaminases, altered liver histology, and increased mRNA expression of endoplasmic reticulum stress marker (Ddit3) and oxidative stress marker (Nqo1). …


Insm1 Governs A Neuronal Progenitor State That Drives Glioblastoma In A Human Stem Cell Model, Patrick A Desouza, Matthew Ishahak, Xuan Qu, Colin Mccornack, Devi Annamalai, Diane D Mao, Rajanikanth Vangipurapu, Yiwei Fu, Alexandre T Vessoni, Ryan T Cleary, Rowland H Han, Punn Augsornworawat, Timothy Woodiwiss, Darby Agovino, Braxton Sizemore, Jessica Kline, Maryam Borhani-Haghighi, Hao Chen, Sangami Pugazenthi, Hiroko Yano, Ting Wang, Luis F Z Batista, Jeffrey R Millman, Albert H Kim Dec 2025

Insm1 Governs A Neuronal Progenitor State That Drives Glioblastoma In A Human Stem Cell Model, Patrick A Desouza, Matthew Ishahak, Xuan Qu, Colin Mccornack, Devi Annamalai, Diane D Mao, Rajanikanth Vangipurapu, Yiwei Fu, Alexandre T Vessoni, Ryan T Cleary, Rowland H Han, Punn Augsornworawat, Timothy Woodiwiss, Darby Agovino, Braxton Sizemore, Jessica Kline, Maryam Borhani-Haghighi, Hao Chen, Sangami Pugazenthi, Hiroko Yano, Ting Wang, Luis F Z Batista, Jeffrey R Millman, Albert H Kim

2020-Current year OA Pubs

Glioblastoma is a lethal brain cancer marked by functional plasticity driven by tumor cell-intrinsic mutations and their interplay with developmental programs. To investigate how canonical glioblastoma mutations promote functional plasticity, we have developed an isogenic human neural stem cell (NSC) model of glioblastoma by sequential addition of TERT promoter, TP53, and PDGFRA point mutations. TP53 loss-of-function increases TERT expression during serial mutagenesis, but only triple mutant NSCs reliably form lethal brain tumors in vivo that recapitulate glioblastoma. Tumor cell evolution triggers stress-related metabolic changes and transitions toward a neuronal progenitor network driven by transcription factor INSM1. INSM1 is highly expressed …


Personalized Perioperative Opioid Strategies In Children: Focus On Methadone, Pharmacogenomics And Prevention Of Persistent Postoperative Opioid Use, Hamsa Priya Bhuchakra, Sennaraj Balasubramanian, Alivia G Nair, Isabella Marcos, Victoria Chen Falconett, Dominic Falcon, Ayesha Abdul Bari, Senthilkumar Sadhasivam Dec 2025

Personalized Perioperative Opioid Strategies In Children: Focus On Methadone, Pharmacogenomics And Prevention Of Persistent Postoperative Opioid Use, Hamsa Priya Bhuchakra, Sennaraj Balasubramanian, Alivia G Nair, Isabella Marcos, Victoria Chen Falconett, Dominic Falcon, Ayesha Abdul Bari, Senthilkumar Sadhasivam

2020-Current year OA Pubs

Persistent postoperative opioid use (PPOU) is an emerging challenge in pediatric perioperative care, with rates as high as 4.7% in opioid-naive adolescents. Despite advances in multimodal analgesia, current protocols often fail to prevent long-term opioid exposure, particularly after high-risk surgeries such as spinal fusions. While multiple strategies exist to reduce PPOU in children, including regional anesthesia and non-opioid analgesics, this review specifically focuses on methadone and pharmacogenomic-guided opioid prescribing as promising approaches. Methadone, a long-acting opioid with mu-opioid agonism, NMDA antagonism, and monoamine reuptake inhibition, has shown encouraging outcomes in adult and emerging pediatric studies but remains underutilized due to …


Synergistic Potential Of Sipuleucel-T In Enhancing Immunotherapy For Metastatic Castration-Resistant Prostate Cancer, Kavita Rawat, Varnika Punia, Parker Mathews, Sara Mccoy, Wilbur Song, Muhammad A Saeed, Russell K Pachynski Dec 2025

Synergistic Potential Of Sipuleucel-T In Enhancing Immunotherapy For Metastatic Castration-Resistant Prostate Cancer, Kavita Rawat, Varnika Punia, Parker Mathews, Sara Mccoy, Wilbur Song, Muhammad A Saeed, Russell K Pachynski

2020-Current year OA Pubs

Clinical trials of immunotherapy in metastatic castration-resistant prostate cancer (mCRPC) have largely been unsuccessful despite promising preclinical studies and proven efficacy in other solid tumors. These disappointing clinical outcomes have been attributed to an immunosuppressive tumor microenvironment, a relative lack of infiltrating immune effector cells, and tumor-related and host-related factors, which collectively render prostate cancer a relatively immunologically "cold" tumor. Sipuleucel-T (Provenge), an autologous cellular immunotherapy, induces an immune response targeted against prostatic acid phosphatase. It received approval from the US Food and Drug Administration in 2010, marking the first immunotherapy to show an overall survival benefit in patients with …


Patient-Centered Televisit For Chronic Obstructive Pulmonary Disease Discharge Transitions: User-Centered Design Study, Joanna Abraham, Alicia Meng, Nicolas Caravelli, Leah Traeger, May Nguyen, Vineet Arora, Valerie G Press Dec 2025

Patient-Centered Televisit For Chronic Obstructive Pulmonary Disease Discharge Transitions: User-Centered Design Study, Joanna Abraham, Alicia Meng, Nicolas Caravelli, Leah Traeger, May Nguyen, Vineet Arora, Valerie G Press

2020-Current year OA Pubs

BACKGROUND: Chronic obstructive pulmonary disease (COPD) affects approximately 16 million Americans and often results in avoidable readmissions due, in part, to medication errors and lack of education. Telehealth interventions can support medication reconciliation and inhaler education following hospital discharge for patients with COPD.

OBJECTIVE: This study aimed to design and prototype TELE-TOC (Telehealth Education: Leveraging Electronic Transitions of Care), a post-discharge, in-home, televisit intervention, and to map its workflow to ensure integration into the routine discharge care transition process for patients with COPD.

METHODS: A user-centered design approach across 3 phases was followed to develop and prototype TELE-TOC. Participants included …


Optimizing Clinical Monitoring And Management Guidelines For Capivasertib In Hr-Positive/Her2-Negative Advanced Breast Cancer: Expert Opinion, Neil M Iyengar, Joyce A O'Shaughnessy, Heather N Moore, Erika Hamilton, Komal L Jhaveri, Cynthia X Ma, Karen Tedesco, Monali Vasekar, Sarah Donahue, Hope S Rugo Dec 2025

Optimizing Clinical Monitoring And Management Guidelines For Capivasertib In Hr-Positive/Her2-Negative Advanced Breast Cancer: Expert Opinion, Neil M Iyengar, Joyce A O'Shaughnessy, Heather N Moore, Erika Hamilton, Komal L Jhaveri, Cynthia X Ma, Karen Tedesco, Monali Vasekar, Sarah Donahue, Hope S Rugo

2020-Current year OA Pubs

Capivasertib, an AKT inhibitor, approved in combination with fulvestrant for hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced breast cancer with ≥1 PIK3CA, AKT1, and/or PTEN alterations, significantly improved progression-free survival in the CAPItello-291 phase 3 trial. However, capivasertib-associated adverse events of diarrhea, rash, and hyperglycemia may require proactive management. This article provides practical recommendations to support prevention and early intervention to optimize adherence and treatment outcomes.


Brain Metabolic-Functional (De)Coupling From Health To Glioma Dysfunction, G Vallini, G Baron, E Silvestri, T Volpi, A G Vlassenko, M S Goyal, A Chiuso, D Cecchin, M Corbetta, A Bertoldo Dec 2025

Brain Metabolic-Functional (De)Coupling From Health To Glioma Dysfunction, G Vallini, G Baron, E Silvestri, T Volpi, A G Vlassenko, M S Goyal, A Chiuso, D Cecchin, M Corbetta, A Bertoldo

2020-Current year OA Pubs

The interplay between brain metabolism and function supports the brain's adaptive capacity in cognitively demanding processes. Prior work has linked glucose metabolism to resting-state fMRI activity, but often overlooks both hemodynamic confounders in the BOLD signal and the brain's dynamic nature. To address this, we employed a novel effective connectivity decomposition, separating symmetric partial covariance, capturing "true" statistical dependencies between regions, from antisymmetric differential covariance, reflecting directional brain flow. In 42 healthy subjects, we show that partial covariance corresponds to metabolic connectivity across regions, while node directionality relates to standardized uptake value ratio, a proxy for local glucose consumption. We …


Comparing Isocitrate Dehydrogenase Inhibitors With Procarbazine, Lomustine, And Vincristine Chemotherapy For Oligodendrogliomas, Gerardo Duran, Diego Pichardo-Rojas, Ahmed Hashim Ali, Peter Passias, Angela Downes, Wilson Z Ray, Gregory J Zipfel, Hakeem J Shakir, Andrew Bauer, Andrew Jea, Ian F Dunn, Jeffrey A Zuccato, Christopher S Graffeo, M Burhan Janjua Dec 2025

Comparing Isocitrate Dehydrogenase Inhibitors With Procarbazine, Lomustine, And Vincristine Chemotherapy For Oligodendrogliomas, Gerardo Duran, Diego Pichardo-Rojas, Ahmed Hashim Ali, Peter Passias, Angela Downes, Wilson Z Ray, Gregory J Zipfel, Hakeem J Shakir, Andrew Bauer, Andrew Jea, Ian F Dunn, Jeffrey A Zuccato, Christopher S Graffeo, M Burhan Janjua

2020-Current year OA Pubs

The abstract has been submitted for presentation to the AANS 2026 meeting being held in San Antonio, TX, USA.


Functional Classification Of Platelet Gene Variants Using Crispr Hdr In Cd34+ Cell-Derived Megakaryocytes, Yasuhiro Kosaka, Brandon Lopez, Nina Kishimoto, Shancy Jacob, Emilie Montenont, Rodrigo Huallanca, Graeson Coughenour, Jorge Di Paola, Justyne Ross, Kristy Lee, Matthew T Rondina, Paul F Bray, Jesse W Rowley Dec 2025

Functional Classification Of Platelet Gene Variants Using Crispr Hdr In Cd34+ Cell-Derived Megakaryocytes, Yasuhiro Kosaka, Brandon Lopez, Nina Kishimoto, Shancy Jacob, Emilie Montenont, Rodrigo Huallanca, Graeson Coughenour, Jorge Di Paola, Justyne Ross, Kristy Lee, Matthew T Rondina, Paul F Bray, Jesse W Rowley

2020-Current year OA Pubs

The interpretation of genetic variants in inherited diseases, such as inherited platelet disorders (IPDs), remains a major clinical challenge, as most are classified as variants of uncertain significance (VUSs). A key barrier to functional evaluation is the lack of accessible, lineage-appropriate assays that reliably reflect native gene regulation and cell-specific biology. To address this gap, we developed CRIMSON HD (CRISPR-edited megakaryocytes [MKs] for surveying platelet variant functions through homology-directed repair [HDR]), a CRISPR-Cas9 HDR-based genome-editing platform applicable to CD34


Enhancing Intergenerational Health In Nigeria: Peripartum As Critical Life Stage For Cardiovascular Health (Enhance-Cvh) Study: Findings From Pre-Implementation Using The Updated Consolidated Framework For Implementation Research (Cfir 2.0), Julia D López, Cecilia Nartey, Debra Haire-Joshu, Victor G Dávila-Román, Mark D Huffman, Clementina E Okoro, Et Al. Dec 2025

Enhancing Intergenerational Health In Nigeria: Peripartum As Critical Life Stage For Cardiovascular Health (Enhance-Cvh) Study: Findings From Pre-Implementation Using The Updated Consolidated Framework For Implementation Research (Cfir 2.0), Julia D López, Cecilia Nartey, Debra Haire-Joshu, Victor G Dávila-Román, Mark D Huffman, Clementina E Okoro, Et Al.

2020-Current year OA Pubs

BACKGROUND: Pregnancy is a crucial period to improve cardiovascular health (CVH) for mothers and their families. The current study emphasizes framework-guided factors that influence the adaptation of an evidence-based intervention (Parents as Teachers and Healthy Eating Active Living Taught at Home [PAT + HEALTH]) to support healthy gestational weight gain and postpartum weight management among pregnant women with obesity and their infants in Nigeria.

METHODS: From May to June 2023, 43 in-depth interviews were conducted with 11 parents, 15 community health extension workers (CHEWs)/health educators, and 17 policymakers/ healthcare supervisors in the Federal Capital Territory, Nigeria. Additionally, nine focus groups …


Operationalizing Passive Sensors Into Scalable, Reproducible, Neurobehavioral Digital Markers For Alzheimer's Disease: Lessons Learned Over 10 Years, Ganesh M Babulal, Matthew Blake, Chen Chen, Yiqi Zhu, Subrata Pal, David C Brown Dec 2025

Operationalizing Passive Sensors Into Scalable, Reproducible, Neurobehavioral Digital Markers For Alzheimer's Disease: Lessons Learned Over 10 Years, Ganesh M Babulal, Matthew Blake, Chen Chen, Yiqi Zhu, Subrata Pal, David C Brown

2020-Current year OA Pubs

No abstract provided.


Resolving The Mysteries Of Brain Clearance And Immune Surveillance, Jonathan Kipnis, Et Al. Dec 2025

Resolving The Mysteries Of Brain Clearance And Immune Surveillance, Jonathan Kipnis, Et Al.

2020-Current year OA Pubs

Recent advances are transforming our understanding of brain fluid dynamics, highlighting cerebrospinal fluid (CSF) flow as a key process in brain waste clearance. While debates persist regarding its anatomical pathways and regulatory mechanisms, certain core principles have become widely accepted. CSF influx occurs primarily along periarterial spaces, solute clearance follows multiple routes, and glymphatic and meningeal lymphatic systems are functionally connected. Brain clearance is influenced by vascular pulsation, neural activity, and sleep. Immune cells at brain-border niches both monitor solute efflux and modulate flow, linking brain clearance to neuroimmune interactions. A deeper understanding of brain clearance could unlock new therapeutic …


Proposed Medical School Curricula For 3d Printing, Danielle Heiser, David H Ballard, Et Al. Dec 2025

Proposed Medical School Curricula For 3d Printing, Danielle Heiser, David H Ballard, Et Al.

2020-Current year OA Pubs

BACKGROUND: 3D printing is increasingly utilized in medical education, providing a hands-on approach to anatomical learning, surgical planning, and interdisciplinary collaboration. Despite growing interest, standardized curricula incorporating 3D printing into medical education are lacking.

METHODS: A multidisciplinary team of medical educators, engineers, and students developed three distinct curricular models to integrate 3D printing into undergraduate medical education. These models include (1) integration into anatomy coursework, (2) a fourth-year clerkship elective, and (3) a pre-clerkship elective. The outline for each curriculum was designed to be adaptable across institutions, emphasizing hands-on learning, imaging segmentation, basic elements of computer-aided design (CAD), and 3D …


Using Gnotobiotic Mice To Decipher Effects Of Gut Microbiome Repair In Undernourished Children On Tuft And Goblet Cell Function, Yi Wang, Hao-Wei Chang, Jiye Cheng, Daniel M Webber, Hannah M Lynn, Matthew C Hibberd, Clara Kao, Ishita Mostafa, Tahmeed Ahmed, Michael J Barratt, Jeffrey I Gordon Dec 2025

Using Gnotobiotic Mice To Decipher Effects Of Gut Microbiome Repair In Undernourished Children On Tuft And Goblet Cell Function, Yi Wang, Hao-Wei Chang, Jiye Cheng, Daniel M Webber, Hannah M Lynn, Matthew C Hibberd, Clara Kao, Ishita Mostafa, Tahmeed Ahmed, Michael J Barratt, Jeffrey I Gordon

2020-Current year OA Pubs

Studies have implicated perturbations in the postnatal development of the gut microbiome as a contributing factor to childhood undernutrition. Compared to a standard ready-to-use supplementary food, a microbiome-directed complementary food (MDCF-2) designed to repair these perturbations produced superior improvements in ponderal and linear growth in clinical trials of Bangladeshi children with moderate acute malnutrition. Here, "reverse translation" experiments are performed where intact fecal microbiomes collected from trial participants before and at the end of treatment are introduced into female gnotobiotic mice just after delivery of their pups. Pups received diets designed to resemble those consumed by children in the trials …


Statistically Significant Association Does Not Imply Improvement In Prediction Of Clinical Outcomes, Shu Jiang, Bernard A Rosner, Graham A Colditz Dec 2025

Statistically Significant Association Does Not Imply Improvement In Prediction Of Clinical Outcomes, Shu Jiang, Bernard A Rosner, Graham A Colditz

2020-Current year OA Pubs

UNLABELLED: In the current landscape of clinical studies, the concept of statistically significant association is often mixed up with the expectation of improved prediction performance. We discuss the two concepts, association and prediction, and present the epidemiologic principles and statistical constructs that underlie the discrepancy between statistically significant associations and the rationale for their lack of impact on improving prediction in terms of discrimination. This issue is illustrated using an existing breast cancer dataset. The concept of statistically significant association should not be mixed up with the expectation of improved discrimination performance. Although some markers may not markedly improve discrimination, …


Fucosylated N-Glycan Landscape Of Triple-Negative Breast Cancer, Danielle A Scott, Souvik Seal, Laura Spruill, Jaclyn Dunne, Graham Colditz, Anand S Mehta, Richard R Drake, Marvella E Ford, Peggi M Angel Dec 2025

Fucosylated N-Glycan Landscape Of Triple-Negative Breast Cancer, Danielle A Scott, Souvik Seal, Laura Spruill, Jaclyn Dunne, Graham Colditz, Anand S Mehta, Richard R Drake, Marvella E Ford, Peggi M Angel

2020-Current year OA Pubs

UNLABELLED: Triple-negative breast cancer (TNBC) is an aggressive subtype of breast cancer lacking targeted therapies. Although aberrant N-glycosylation is a hallmark of malignancy, the specific roles of core-fucosylated (CF) and outer arm-fucosylated (OAF) N-glycans in TNBC progression and patient survival remain underexplored. This study utilized multiplexed glycomics by matrix-assisted laser desorption/ionization imaging mass spectrometry to spatially profile 348 N-glycans in 59 TNBC tumors with clinical characteristics including survival data. Spatial analysis revealed distinct localization patterns of CF and OAF isomers within tumor microenvironments. Strikingly, OAF glycans, but not CF, were strongly associated with tumor stage, with expression increasing from stage …


Molecular Response To The Non-Lytic Peptide Bac7 (1-35) Triggers Disruption Of Klebsiella Pneumoniae Biofilm, Robert L Beckman I V, Berta Martinez, Flor Z. Santiago, Gabriela N. Echeverria, Bruno V. Pinheiro, Marcelo D. T. Torres, Logan Suits, Shantal Garcia, Paeton L. Wantuch, Cesar De La Fuente-Nunez, Prahathees Eswara, David A. Rosen, Renee M. Fleeman Dec 2025

Molecular Response To The Non-Lytic Peptide Bac7 (1-35) Triggers Disruption Of Klebsiella Pneumoniae Biofilm, Robert L Beckman I V, Berta Martinez, Flor Z. Santiago, Gabriela N. Echeverria, Bruno V. Pinheiro, Marcelo D. T. Torres, Logan Suits, Shantal Garcia, Paeton L. Wantuch, Cesar De La Fuente-Nunez, Prahathees Eswara, David A. Rosen, Renee M. Fleeman

2020-Current year OA Pubs

Klebsiella pneumoniae is becoming increasingly difficult to treat as multidrug-resistant (MDR) strains become more prevalent. The formation of biofilm heightens this threat by embedding bacterial cells in a polysaccharide-rich matrix that limits antibiotic penetration. Here we dissect the anti-biofilm bovine host-defense cathelicidin peptide fragment bac7 (1-35), exploring its anti-biofilm mechanism, evaluating its ability to curb colonization of the vital organs by hypervirulent K. pneumoniae, and testing its breadth of activity against diverse clinical isolates. Transcriptomic profiling revealed that bac7 (1-35) simultaneously compromises the bacterial membrane and inhibits ribosomal function, a dual assault that precipitates rapid biofilm collapse and blocks bacterial …


The Non-Communicable Disease E-Cohort For Hypertension In Latin America: Concept, Survey Development And Study Protocol, Patricia J Garcia, Laura Espinoza-Pajuelo, Hannah H Leslie, Margaret E Kruk, Catherine Arsenault, Agustina Mazzoni, Jesus Medina-Ranilla, Javier Roberti, Marina Guglielmino, Ezequiel Garcia-Elorrio Dec 2025

The Non-Communicable Disease E-Cohort For Hypertension In Latin America: Concept, Survey Development And Study Protocol, Patricia J Garcia, Laura Espinoza-Pajuelo, Hannah H Leslie, Margaret E Kruk, Catherine Arsenault, Agustina Mazzoni, Jesus Medina-Ranilla, Javier Roberti, Marina Guglielmino, Ezequiel Garcia-Elorrio

2020-Current year OA Pubs

Non-communicable diseases like hypertension cause substantial morbidity and mortality in low- and middle-income countries, where limited access to high-quality care contributes to millions of preventable deaths annually. Traditional assessments of health system performance often rely on structural indicators and cross-sectional, overlooking patient experiences and care processes. In Latin America, amid rising cardiovascular disease, longitudinal tools are needed to guide improvements in healthcare delivery models, particularly for chronic diseases such as hypertension. The NCD e-Cohort is a prospective, longitudinal, mixed-mode (in-person and phone) survey designed to assess health system quality in Latin America. Developed by the QuEST Network, it integrates validated …


A Roadmap To Scale Up Person-Centred Care In The Hiv Response: Recommendations From A Global Consensus-Building Process, Lina Golob, Elvin H Geng, Et Al. Dec 2025

A Roadmap To Scale Up Person-Centred Care In The Hiv Response: Recommendations From A Global Consensus-Building Process, Lina Golob, Elvin H Geng, Et Al.

2020-Current year OA Pubs

INTRODUCTION: World Health Organization global normative guidance recommends person-centred care (PCC) approaches to reduce HIV-related mortality and morbidity and to improve health-related quality of life (HrQoL). However, consensus on the priority PCC elements and guidance on how different stakeholders can realize PCC principles at the health systems, service delivery and individual client-healthcare worker (HCW) levels are lacking. We conducted a global consensus-building process to define core PCC elements and develop recommendations for implementation at scale.

METHODS: We used a multi-phase process to build consensus and prioritize recommendations, consisting of a literature review, five stakeholder consultations (34-43 participants each) between July …


Clinical Characteristics And Real-World Outcomes In Patients With Mucopolysaccharidosis Ii Over 18 Years: Final Report Of The Hunter Outcome Survey, Joseph Muenzer, Jaco Botha, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Christoph Kampmann, Bianca Link, Shuan-Pei Lin, David Molter, Julian Raiman, Maurizio Scarpa, Anna Tylki-Szymańska, Siddharth Jain, David A H Whiteman, Barbara K Burton Dec 2025

Clinical Characteristics And Real-World Outcomes In Patients With Mucopolysaccharidosis Ii Over 18 Years: Final Report Of The Hunter Outcome Survey, Joseph Muenzer, Jaco Botha, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Christoph Kampmann, Bianca Link, Shuan-Pei Lin, David Molter, Julian Raiman, Maurizio Scarpa, Anna Tylki-Szymańska, Siddharth Jain, David A H Whiteman, Barbara K Burton

2020-Current year OA Pubs

BACKGROUND: Mucopolysaccharidosis II (MPS II) is a rare, progressive, X-linked lysosomal storage disease. Enzyme replacement therapy (ERT) with intravenous (IV) idursulfase has been approved for the treatment of patients with MPS II since 2005. The Hunter Outcome Survey (HOS; NCT03292887) was established as a condition of approval to monitor the long-term safety and effectiveness of idursulfase. Here, we report the final results from HOS.

METHODS: HOS was a multicenter, long-term, observational registry that enrolled patients with a biochemically and/or genetically confirmed diagnosis of MPS II who were untreated or treated with idursulfase and/or bone marrow transplant. Patients were enrolled prospectively …


Current Practices And Trends In Surgical Decision-Making For Children With Lennox-Gastaut Syndrome: A Cross-Sectional Survey By The Pediatric Epilepsy Research Consortium, Michelle Y Chiu, Renée A Shellhaas, Et Al. Dec 2025

Current Practices And Trends In Surgical Decision-Making For Children With Lennox-Gastaut Syndrome: A Cross-Sectional Survey By The Pediatric Epilepsy Research Consortium, Michelle Y Chiu, Renée A Shellhaas, Et Al.

2020-Current year OA Pubs

OBJECTIVE: The objective of this study is to characterize contemporary surgical management practices and factors influencing surgical decision-making in the management of Lennox-Gastaut Syndrome (LGS) across pediatric epilepsy centers in the United States.

METHODS: A 45-item cross-sectional survey was developed and distributed to Pediatric Epilepsy Research Consortium centers. Domains included institutional demographics, surgical evaluation timing and goals, presurgical workup, procedural utilization and outcomes, and barriers to surgery. Descriptive statistics and thematic analysis were performed.

RESULTS: Thirty-two pediatric epilepsy centers participated (38% response), the majority comprising pediatric epileptologists at Level 4 National Association of Epilepsy Centers. Most centers (66%) considered surgery …


Association Of Area Deprivation Index With Perinatal And Covid-19 Outcomes During The Early Pandemic: A Secondary Analysis Of A Prospective Cohort Study, Abigail Bell, Megan Foeller, Candice Woolfolk, Emily Diveley, Fan Zhang, Amy Scheffer, Ruizhi Huang, Daniel Jackson, Nandini Raghuraman, Ebony Carter, Indira U Mysorekar, Jeannie C Kelly Dec 2025

Association Of Area Deprivation Index With Perinatal And Covid-19 Outcomes During The Early Pandemic: A Secondary Analysis Of A Prospective Cohort Study, Abigail Bell, Megan Foeller, Candice Woolfolk, Emily Diveley, Fan Zhang, Amy Scheffer, Ruizhi Huang, Daniel Jackson, Nandini Raghuraman, Ebony Carter, Indira U Mysorekar, Jeannie C Kelly

2020-Current year OA Pubs

OBJECTIVE: We sought to evaluate the relationship between neighborhood-level deprivation and perinatal outcomes, particularly preterm birth, as well as COVID-19 outcomes among pregnant patients during the early pandemic.

METHODS: We performed a secondary analysis of a prospective longitudinal cohort study that investigated the impact of prenatal COVID-19 exposure on perinatal outcomes. Pregnant patients were recruited 12/23/20-7/18/22 and serially assessed for COVID-19 exposure during pregnancy, with serum antibody testing, electronic medical record review, and patient report. For this secondary analysis, address at the time of delivery was used to categorize patients into low ADI (≤50

RESULTS: 306 patients were included: 124 …


Impact Of Silencing Automated Penicillin Cross-Reactivity Alerts On Perioperative Antibiotic Prescribing And Surgical Site Infection Rates, Michael J Durkin, Joshua Nordman, Alice Bewley, Andrew Atkinson, Jonas Marschall, Helen Newland, Kimberly G Blumenthal Dec 2025

Impact Of Silencing Automated Penicillin Cross-Reactivity Alerts On Perioperative Antibiotic Prescribing And Surgical Site Infection Rates, Michael J Durkin, Joshua Nordman, Alice Bewley, Andrew Atkinson, Jonas Marschall, Helen Newland, Kimberly G Blumenthal

2020-Current year OA Pubs

We evaluated the impact of silencing penicillin cross-reactivity alerts on perioperative antibiotic prescribing and surgical site infections (SSIs) in 6 hospitals using an interrupted time series analysis. Silencing the alerts minimally increased cefazolin prescribing among penicillin allergy labeled patients (sensitivity analysis only;


Assessment Of Knowledge And Attitudes Of Undergraduate Students On Trichomoniasis In Ghana, Christopher Yaw Dumevi, Ezekiel Kofi Vicar, Lorie Christla Quansah, Rhoda Oklu, Smith Joshua, Joyce Junior Asiamah, James-Paul Kretchy, George Boateng Kyei, Patience B Tetteh-Quarcoo, Nicholas T K D Dayie, Irene Ayi, Patrick F Ayeh-Kumi Dec 2025

Assessment Of Knowledge And Attitudes Of Undergraduate Students On Trichomoniasis In Ghana, Christopher Yaw Dumevi, Ezekiel Kofi Vicar, Lorie Christla Quansah, Rhoda Oklu, Smith Joshua, Joyce Junior Asiamah, James-Paul Kretchy, George Boateng Kyei, Patience B Tetteh-Quarcoo, Nicholas T K D Dayie, Irene Ayi, Patrick F Ayeh-Kumi

2020-Current year OA Pubs

BACKGROUND: Trichomoniasis is a non-viral neglected sexually transmitted disease of public health importance. Undergraduate students, often experiencing newfound freedom from family oversight, may engage in risky sexual behaviors, predisposing them to trichomoniasis and other sexually transmitted infections (STIs). This study aimed to assess the knowledge, attitudes, and practices towards trichomoniasis and associated factors among the Central University students in the Greater Accra Region of Ghana.

METHODS: An institutional cross-sectional study was conducted between March and July 2024, involving 387 undergraduate students from Central University who were selected using a simple random sampling technique. Data were collected using a structured researcher-led …


Functional Connectivity Associations With Markers Of Disease Progression In Grn Pathogenic Variant Carriers, Taru M Flagan, Nupur Ghoshal, Et Al. Dec 2025

Functional Connectivity Associations With Markers Of Disease Progression In Grn Pathogenic Variant Carriers, Taru M Flagan, Nupur Ghoshal, Et Al.

2020-Current year OA Pubs

OBJECTIVE: Autosomal dominant progranulin (GRN) pathogenic variants are a genetic cause of frontotemporal lobar degeneration. Though clinical trials for GRN-related therapies are underway, there is an unmet need for biomarkers that can predict symptom onset and track disease progression. We previously showed that asymptomatic GRN variant carriers exhibit thalamocortical hyperconnectivity that increases with age, presumably as they are approaching symptom onset. Whether hyperconnectivity arises concomitantly with markers of neurodegeneration remains unclear.

METHODS: Utilizing T1 and task-free functional magnetic resonance imaging from 49 asymptomatic and 26 symptomatic GRN variant carriers, we determined the relationships between functional connectivity, as measured by voxelwise …


A Guide For Initiating And Managing Chimeric Antigen Receptor T Cell Therapy Clinical Trials In Autoimmune Rheumatic Diseases, Roberto Caricchio, Stacie Bell, Sasha Bernatsky, Maria Dall'era, David Goddard, Kenneth Kalunian, Alfred H J Kim, Fotios Koumpouras, Jose Rubio, Amit Saxena, Saira Sheikh, Lupus Clinical Investigators Network Dec 2025

A Guide For Initiating And Managing Chimeric Antigen Receptor T Cell Therapy Clinical Trials In Autoimmune Rheumatic Diseases, Roberto Caricchio, Stacie Bell, Sasha Bernatsky, Maria Dall'era, David Goddard, Kenneth Kalunian, Alfred H J Kim, Fotios Koumpouras, Jose Rubio, Amit Saxena, Saira Sheikh, Lupus Clinical Investigators Network

2020-Current year OA Pubs

Chimeric antigen receptor (CAR) T-cell therapy, long transformative in oncology, is now rapidly emerging as a frontier in autoimmune rheumatic diseases, particularly systemic lupus erythematosus (SLE), driven by accumulating evidence of deep B-cell depletion, immune "resetting," and durable drug-free remission in early studies, yet its translation into rheumatology demands mastery of formidable logistical, regulatory, clinical, and ethical complexities that span institutional readiness, multidisciplinary team formation, stringent regulatory compliance, sophisticated operational workflows, comprehensive patient selection and education, meticulous clinical management of both classical toxicities (CRS, ICANS, ICAHT) and autoimmune-specific reactions such as LICATS, robust financial and resource planning, and long-term follow-up …


Improved Accuracy For Myocardial Blood Flow Mapping With Deep Learning-Enabled Cmr Arterial Spin Labeling (Deepmasl): Validation By Microsphere In Vivo, Ran Li, Caleb Berberet, Qi Huang, Pamela K Woodard, Jie Zheng Dec 2025

Improved Accuracy For Myocardial Blood Flow Mapping With Deep Learning-Enabled Cmr Arterial Spin Labeling (Deepmasl): Validation By Microsphere In Vivo, Ran Li, Caleb Berberet, Qi Huang, Pamela K Woodard, Jie Zheng

2020-Current year OA Pubs

BACKGROUND: Current myocardial arterial spin labeling (ASL) methods are sensitive to noise (background and physiology), which limits the accuracy of myocardial blood flow (MBF) measurement. In this study, we demonstrated a new deep learning-enabled myocardial ASL approach (DeepMASL) and evaluated its accuracy to quantify MBF in a canine model of coronary arterial disease in vivo. The reference method was invasive microsphere measurements.

METHODS: Eighteen mongrel dogs were divided into two groups: healthy (n = 9) and coronary stenosis (n = 9). The latter was induced in an open-chest model with 3 types of stenosis: 50% (n= 3), 70% (n = …


Buprenorphine Treatment During The Extended Postpartum Period: A Survey Of Patient Experiences, Bridget Galati, Heather Jacobsen, Melissa Krauss, Melissa Mills, Brittaney Vaughn, Sam Williams, Michael Wenzinger, Cynthia Rogers, Nandini Raghuraman, Jeannie Kelly Dec 2025

Buprenorphine Treatment During The Extended Postpartum Period: A Survey Of Patient Experiences, Bridget Galati, Heather Jacobsen, Melissa Krauss, Melissa Mills, Brittaney Vaughn, Sam Williams, Michael Wenzinger, Cynthia Rogers, Nandini Raghuraman, Jeannie Kelly

2020-Current year OA Pubs

OBJECTIVE: The late postpartum period, defined as greater than 6 weeks to 12 months, is a high-risk time for individuals with opioid use disorder (OUD). Discontinuation rates of buprenorphine treatment for OUD during this period are high. Little is known about patients' subjective experience with buprenorphine treatment during this time. We sought to assess patient treatment experience, goals, facilitators and barriers to continued buprenorphine treatment throughout the extended postpartum period.

METHODS: We performed a cross-sectional survey of postpartum patients who received buprenorphine treatment, including sublingual and long-acting injectable formulations, in an interdisciplinary, wrap-around clinic for maternal OUD between February 2022 …


Does Health Insurance Coverage Improve Cardiometabolic Risk Factor Levels? Quasi-Experimental Evidence From India, Kavita Singh, Anubha Agarwal, Mark D Huffman, Et Al. Dec 2025

Does Health Insurance Coverage Improve Cardiometabolic Risk Factor Levels? Quasi-Experimental Evidence From India, Kavita Singh, Anubha Agarwal, Mark D Huffman, Et Al.

2020-Current year OA Pubs

BACKGROUND: Chronic conditions cause notable health and economic burdens. While health insurance enables access to healthcare, its effects on chronic care outcomes remain under-explored.

OBJECTIVE: To examine the association between health insurance coverage and cardiometabolic risk factors among people with chronic conditions in India.

METHODS: Data from the Centre for Cardiometabolic Risk Reduction in South Asia (CARRS) and Solan studies, including 2,926 adults with chronic conditions were analyzed using propensity score weighting to evaluate the associations between health insurance and cardiometabolic risk factors (HbA1c, low-density lipoprotein cholesterol [LDLc], and blood pressure [BP]) and self-reported health status (measured using European Quality …


Cholinergic Dysfunction In Occupational Manganese Exposure, T Noah Hutson, Susan Searles Nielsen, Natalie Senini, John O'Donnell, Hubert P Flores, Tamara Hershey, Joel S Perlmutter, Anil Kumar Soda, Stephen M Moerlein, Zhude Tu, Michael Kasper, Lianne Sheppard, Brad A Racette, Susan R Criswell Dec 2025

Cholinergic Dysfunction In Occupational Manganese Exposure, T Noah Hutson, Susan Searles Nielsen, Natalie Senini, John O'Donnell, Hubert P Flores, Tamara Hershey, Joel S Perlmutter, Anil Kumar Soda, Stephen M Moerlein, Zhude Tu, Michael Kasper, Lianne Sheppard, Brad A Racette, Susan R Criswell

2020-Current year OA Pubs

BACKGROUND AND OBJECTIVE: Excessive exposure to manganese (Mn) produces a clinical syndrome of parkinsonism and cognitive impairment. However, our understanding of the mechanisms of Mn neurotoxicity remains limited. This study aimed to evaluate the relationships between Mn exposure, cholinergic function, and cognitive impairment in exposed workers.

METHODS: We assessed brain cholinergic function using vesicular acetylcholine transporter (VAChT) radiotracer (-)-(1-(8-(2-[(18)F]fluoroethoxy)-3-hydroxy-1,2,3,4-tetrahydronaphthalen-2-yl)-piperidin-4-yl)(4-fluorophenyl)methanone (VAT) with positron emission tomography (PET) in 21 Mn-exposed workers. We estimated occupational Mn exposure from work histories and the MRI pallidal index. A cognitive control battery consisting of the Verbal Fluency (VF), Letter Number Sequencing (LNS), Two-Back Letter Task (2B), …