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Aav Gene Therapy For Duchenne Muscular Dystrophy: The Embark Phase 3 Randomized Trial, Jerry R Mendell, Craig M Zaidman, Et Al. Jan 2025

Aav Gene Therapy For Duchenne Muscular Dystrophy: The Embark Phase 3 Randomized Trial, Jerry R Mendell, Craig M Zaidman, Et Al.

2020-Current year OA Pubs

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of functional dystrophin, beginning at birth and leading to progressive impaired motor function, loss of ambulation and life-threatening cardiorespiratory complications. Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with DMD. Ambulatory males with DMD, ≥4 years to <8 years of age, were randomized and stratified by age group and North Star Ambulatory Assessment (NSAA) score to single-administration intravenous delandistrogene moxeparvovec (1.33 × 10


Social Anxiety, Negative Affect, And Hearing Difficulties In Adults, Katrina Kate S. Mcclannahan, Sarah Mcconkey, Julia M Levitan, Thomas L Rodebaugh, Jonathan E Peelle Jan 2025

Social Anxiety, Negative Affect, And Hearing Difficulties In Adults, Katrina Kate S. Mcclannahan, Sarah Mcconkey, Julia M Levitan, Thomas L Rodebaugh, Jonathan E Peelle

2020-Current year OA Pubs

Subjective ratings of communication function reflect both auditory sensitivity and the situational, social, and emotional consequences of communication difficulties. Listeners interact with people and their environment differently, have various ways of handling stressful situations, and have diverse communication needs. Therefore, understanding the relationship between auditory and mental health factors is crucial for the holistic diagnosis and treatment of communication difficulty, particularly as mental health and communication function may have bidirectional effects. The goal of this study was to evaluate the degree to which social anxiety and negative affect (encompassing generalized anxiety, depression, and anger) contributed to subjective communication function (hearing …


Recombinant Human Alpha-N-Acetylglucosamine-6-Sulfatase Delivered To Sanfilippo D Mice With Repeated Intracerebroventricular Injections Corrects Cns Pathology, Grant L. Austin, Feng Wang, Steven Q. Le, Alexander Sorensen, Shan Li, Lai C. Foong, Srikanth Singamsetty, Jill Wood, Tsui-Fen Chou, Patricia I. Dickson Jan 2025

Recombinant Human Alpha-N-Acetylglucosamine-6-Sulfatase Delivered To Sanfilippo D Mice With Repeated Intracerebroventricular Injections Corrects Cns Pathology, Grant L. Austin, Feng Wang, Steven Q. Le, Alexander Sorensen, Shan Li, Lai C. Foong, Srikanth Singamsetty, Jill Wood, Tsui-Fen Chou, Patricia I. Dickson

2020-Current year OA Pubs

Mucopolysaccharidosis type IIID (MPS IIID; Sanfilippo D) is caused by biallelic pathogenic variants in N-acetylglucosamine-6-sulfatase (GNS), which participates in catabolism of heparan sulfate (HS) glycosaminoglycans. Characterization of MPS IIID disease at a cellular level has not been robustly achieved. We used unbiased quantitative proteomics to establish a cellular phenotype for MPS IIID mice. Recombinant human GNS (rhGNS), a variant of which previously demonstrated single dose efficacy in MPS IIID human fibroblasts and in MPS IIID neonatal mice, was used to establish a repeat dosing schedule to treat MPS IIID mice. Adult Gns KO mice or heterozygous carriers were treated via …


Thalamic And Visual Network Dysfunction Relates To Tremor Response In Thalamic Deep Brain Stimulation, Aimee E Morris, Babatunde Adeyemo, Meghan C Campbell, Abraham Z Snyder, Joel S Perlmutter, Jonathan W Mink, Scott A Norris Jan 2025

Thalamic And Visual Network Dysfunction Relates To Tremor Response In Thalamic Deep Brain Stimulation, Aimee E Morris, Babatunde Adeyemo, Meghan C Campbell, Abraham Z Snyder, Joel S Perlmutter, Jonathan W Mink, Scott A Norris

2020-Current year OA Pubs

BACKGROUND: Essential tremor (ET) is the most common movement disorder in adults, but its pathogenesis is incompletely understood. Deep brain stimulation of the ventral intermediate thalamic nucleus (VIM DBS) provides effective treatment for medically-refractory cases. We aimed to determine how pre-surgical resting-state functional connectivity (FC) in medically-refractory ET relates to VIM DBS clinical response.

METHODS: We analyzed resting-state FC MRI in 21 participants with medically-refractory ET who subsequently underwent VIM DBS and 34 matched controls. We applied rigorous quality assurance to minimize motion artifact. Whole-brain correlation matrices were computed across 300 cortical, subcortical, and cerebellar regions and compared across groups …


Plasma Nfl And Cognitive Functioning In Older Adults: The Moderating Role Of Hdl Cholesterol, Ramkrishna K Singh, Semere Bekena, Yiqi Zhu, Carlos Cruchaga, Steven E Arnold, Beau M Ances, Ganesh M Babulal Jan 2025

Plasma Nfl And Cognitive Functioning In Older Adults: The Moderating Role Of Hdl Cholesterol, Ramkrishna K Singh, Semere Bekena, Yiqi Zhu, Carlos Cruchaga, Steven E Arnold, Beau M Ances, Ganesh M Babulal

2020-Current year OA Pubs

BACKGROUND AND OBJECTIVES: Plasma neurofilament light chain (NfL) is a marker of neuroaxonal injury associated with cognitive decline. High-density lipoprotein (HDL) cholesterol has neuroprotective properties, but its interaction with neurodegeneration remains unclear. This study examined whether HDL moderates the association between NfL and cognitive performance.

METHODS: Baseline data from 417 participants in the Aging Adult Brain Connectome study were analyzed. Plasma NfL and HDL were measured via Simoa and enzymatic assays; cognition was assessed using Montreal Cognitive Assessment (MoCA) and Preclinical Alzheimer Cognitive Composite (PACC). Generalized linear models were used to evaluate NfL and HDL interactions, adjusting for demographics. Sensitivity …


Enhancing Safety: Multi-Institutional Fmea And Fta On 177 Lu-Based Radio-Pharmaceutical Therapy, Siju C George, Santiago Aguirre, Nichole M Maughan, Ranjini Tolakanahalli, E James Jebaseelan Samuel, Sven L Gallo, Jacqueline E Zoberi, Yongsook C Lee Jan 2025

Enhancing Safety: Multi-Institutional Fmea And Fta On 177 Lu-Based Radio-Pharmaceutical Therapy, Siju C George, Santiago Aguirre, Nichole M Maughan, Ranjini Tolakanahalli, E James Jebaseelan Samuel, Sven L Gallo, Jacqueline E Zoberi, Yongsook C Lee

2020-Current year OA Pubs

PURPOSE: This study investigates potential failure modes and conducts failure mode and effects analysis (FMEA) and fault tree analysis (FTA) on the administration of

METHODS: We reviewed the literature to investigate the administration of Pluvicto and Lutathera, focusing on prospective procedural failures and potential failure modes (PFMs) and their outcomes. We distributed an FMEA survey to multiple experienced centers in

RESULTS: The findings from the literature review and survey responses on the prospective study have identified several critical areas at risk of failure. These areas include non-optimized treatment delivery, inadequate patient monitoring, and lack of safety training, leading to radiation …


Feasibility Of Endobronchial Ultrasound Guided Miniforceps Biopsy For Pd-L1 Expression And Quantitative Tissue Analysis, Max T Wayne, Nathaniel G Moulton, Cody Weimholt, Praveen Chenna, Alexander C Chen Jan 2025

Feasibility Of Endobronchial Ultrasound Guided Miniforceps Biopsy For Pd-L1 Expression And Quantitative Tissue Analysis, Max T Wayne, Nathaniel G Moulton, Cody Weimholt, Praveen Chenna, Alexander C Chen

2020-Current year OA Pubs

BACKGROUND: Testing for targeting programmed death ligand 1 (PD-L1) is standard of care for patients with newly diagnosed non-small cell lung cancer (NSCLC) but is only approved for use with core biopsy specimens. Endobronchial ultrasound guided miniforceps biopsy (EBUS-MFB) is an approach to obtain core biopsy material but data assessing the ability of EBUS-MFB to adequately test for PD-L1 is lacking. We evaluate the feasibility of EBUS-MFB to acquire adequate tissue for PD-L1 testing and look to compare the quality of specimens between EBUS-MFB and endobronchial ultrasound guided transbronchial needle aspiration (EBUS-TBNA) using a standard method of tissue analysis.

METHODS: …


Iliac Crest Bone Block Autograft Transfer For Ballistic Posterior Glenoid Fracture: A Case Report, Jordan Cook Serotte, Hayden Baker, Cody Lee, Jason A Strelzow Jan 2025

Iliac Crest Bone Block Autograft Transfer For Ballistic Posterior Glenoid Fracture: A Case Report, Jordan Cook Serotte, Hayden Baker, Cody Lee, Jason A Strelzow

2020-Current year OA Pubs

No abstract provided.


Chronic Recurrent Multifocal Osteomyelitis-Like Paraneoplastic Syndrome As Initial Presentation Of Nodular Sclerosis Classical Hodgkin Lymphoma, Michel Attieh, Seyed Reza Taha, Selim Nasser, Fouad Boulos Jan 2025

Chronic Recurrent Multifocal Osteomyelitis-Like Paraneoplastic Syndrome As Initial Presentation Of Nodular Sclerosis Classical Hodgkin Lymphoma, Michel Attieh, Seyed Reza Taha, Selim Nasser, Fouad Boulos

2020-Current year OA Pubs

Hodgkin lymphoma (HL) presenting with initial skeletal symptoms as a paraneoplastic phenomenon is extremely rare. Herein, we report the case of a 26-year-old man with nodular sclerosis classical HL (NSCHL) who presented with low back pain as the initial symptom. Imaging studies were unremarkable except for right S1-S2 sacral marrow edema on MRI, and multiple biopsies showed only inflammatory changes, resulting in a tentative diagnosis of chronic recurrent multifocal osteomyelitis. Later in the course of the disease, after performing routine series of MRIs, lymphadenopathy was finally detected. Core biopsies of the axillary and pelvic lymph nodes subsequently confirmed the diagnosis …


Wireless Modular Implantable Neural Device With One-Touch Magnetic Assembly For Versatile Neuromodulation, Inho Kang, John Bilbily, Choong Yeon Kim, Chuanqian Shi, Manish K Madasu, Eun Young Jeong, Kyle E Parker, Do A Kwon, Bum-Joon Jung, Jae-Soon Yang, Juhyun Lee, Noah D L Kabbaj, Wonhee Lee, Jun-Bo Yoon, Ream Al-Hasani, Jianliang Xiao, Jordan G Mccall, Jae-Woong Jeong Jan 2025

Wireless Modular Implantable Neural Device With One-Touch Magnetic Assembly For Versatile Neuromodulation, Inho Kang, John Bilbily, Choong Yeon Kim, Chuanqian Shi, Manish K Madasu, Eun Young Jeong, Kyle E Parker, Do A Kwon, Bum-Joon Jung, Jae-Soon Yang, Juhyun Lee, Noah D L Kabbaj, Wonhee Lee, Jun-Bo Yoon, Ream Al-Hasani, Jianliang Xiao, Jordan G Mccall, Jae-Woong Jeong

2020-Current year OA Pubs

Multimodal neural interfaces open new opportunities in brain research by enabling more sophisticated and systematic neural circuit dissection. Integrating complementary features across distinct functional domains, these multifunctional neural probes have greatly advanced the interrogation of complex neural circuitry. However, introducing multiple functionalities into a compact form factor for freely behaving animals presents substantial design hurdles that complicate the device or require more than one device. Moreover, fixed functionality poses challenges in meeting the dynamic needs of chronic neuroscience inquiry, such as replacing consumable parts like batteries or drugs. To address these limitations, the modular implantable neural device (MIND) is introduced …


Psychosocial Behavioral Phenotypes Of Racially/Ethnically Minoritized Older Adults Enrolled In Habs-Hd Differ On Neuroimaging Measures Of Brain Age Gap, Hippocampal Volume, And Cortical Thickness, Alexandra L Clark, Makenna B Mcgill, Alexandra J Weigand, Julie K Wisch, Kalen Petersen, Beau Ances, Meredith N Braskie, Sid O'Bryant, Kelsey R Thomas, Habs‐Hd Study Team Jan 2025

Psychosocial Behavioral Phenotypes Of Racially/Ethnically Minoritized Older Adults Enrolled In Habs-Hd Differ On Neuroimaging Measures Of Brain Age Gap, Hippocampal Volume, And Cortical Thickness, Alexandra L Clark, Makenna B Mcgill, Alexandra J Weigand, Julie K Wisch, Kalen Petersen, Beau Ances, Meredith N Braskie, Sid O'Bryant, Kelsey R Thomas, Habs‐Hd Study Team

2020-Current year OA Pubs

INTRODUCTION: This study examined whether previously identified psychosocial behavioral phenotypes differed on structural neuroimaging markers.

METHODS: Latent profile analysis (LPA) employed in a sample of 1820 community-dwelling older adults (1118 Hispanic and 702 Black) replicated previous Low Resource/Low Distress, High Resource/Low Distress, and Low Resource/High Distress phenotype classifications. Analyses of covariance (ANCOVAs) adjusting for relevant factors examined phenotype differences on neuroimaging outcomes of predicted brain age gap (BAG) (DeepBrainNet

RESULTS: The Low Resource/Low Distress and Low Resource/High Distress phenotypes had significantly higher predicted BAGs relative to the High Resource/Low Distress phenotype, and the Low Resource/High Distress group displayed significantly lower …


Multi-Echo Acquisition And Thermal Denoising Advances Precision Functional Imaging, Julia Moser, Alyssa K Labonte, Michael J Myers, Babatunde Adeyemo, Abraham Z Snyder, Evan M Gordon, Nico U F Dosenbach, Timothy O Laumann, Chad M Sylvester, Et Al. Jan 2025

Multi-Echo Acquisition And Thermal Denoising Advances Precision Functional Imaging, Julia Moser, Alyssa K Labonte, Michael J Myers, Babatunde Adeyemo, Abraham Z Snyder, Evan M Gordon, Nico U F Dosenbach, Timothy O Laumann, Chad M Sylvester, Et Al.

2020-Current year OA Pubs

The characterization of individual functional brain organization with Precision Functional Mapping has provided important insights in recent years in adults. However, little is known about the ontogeny of inter-individual differences in brain functional organization during human development. Precise characterization of systems organization during periods of high plasticity is likely to be essential for discoveries promoting lifelong health. Obtaining precision functional magnetic resonance imaging (fMRI) data during development has unique challenges that highlight the importance of establishing new methods to improve data acquisition, processing, and analysis. Here, we investigate two methods that can facilitate attaining this goal: multi-echo (ME) data acquisition …


Acute Metabolic Decompensation After Liver Transplant In A Patient With Maple Syrup Urine Disease, Shao Ching Tu, Marium Khan, Katie Wolfe, Sakil S Kulkarni, Elizabeth Toolan, Dorothy K Grange Jan 2025

Acute Metabolic Decompensation After Liver Transplant In A Patient With Maple Syrup Urine Disease, Shao Ching Tu, Marium Khan, Katie Wolfe, Sakil S Kulkarni, Elizabeth Toolan, Dorothy K Grange

2020-Current year OA Pubs

Maple syrup urine disease (MSUD) is an inborn error of metabolism characterized by the accumulation of branched-chain amino acids (leucine, isoleucine, and valine) caused by a defect in the branched-chain alpha-keto acid dehydrogenase complex. Liver transplant is an effective therapy for MSUD, and patients can usually tolerate a regular diet after transplant without symptomatic metabolic decompensation. Most post-transplant patients do not follow a sick-day diet. We report a case of a 7-year-old male with MSUD Type IA, status post-liver transplant at 2 years of age, who presented with profound encephalopathy following poor oral intake and vomiting for 3 days. Broad …


Schistosomiasis Interventions In Africa: Assessment And Systematic Review, Christopher Yaw Dumevi, George Boateng Kyei, Patience B Tetteh-Quarcoo, James-Paul Kretchy, Irene Ayi, Patrick F Ayeh-Kumi Jan 2025

Schistosomiasis Interventions In Africa: Assessment And Systematic Review, Christopher Yaw Dumevi, George Boateng Kyei, Patience B Tetteh-Quarcoo, James-Paul Kretchy, Irene Ayi, Patrick F Ayeh-Kumi

2020-Current year OA Pubs

No abstract provided.


Microbial Degradation Of Persistent Organic Pollutants In Food Supply Chain, Pankaj Kumar, Ajay Singh, Manikant Tripathi, Deepti Diwan, R Manimekalai, Minaxi Sharma, Sudisha Jogajah, Ramesh Chandra Dubey, Gauri Dutt Sharma Jan 2025

Microbial Degradation Of Persistent Organic Pollutants In Food Supply Chain, Pankaj Kumar, Ajay Singh, Manikant Tripathi, Deepti Diwan, R Manimekalai, Minaxi Sharma, Sudisha Jogajah, Ramesh Chandra Dubey, Gauri Dutt Sharma

2020-Current year OA Pubs

No abstract provided.


Qualitative And Quantitative Analysis Of A Novel Dental Health Education Program To Improve Dental Care Utilization By Bhutanese-, Burmese-, And Swahili-Speaking Refugees In A Midwestern United States City, Chandra Swanson, Katherine Gordon, Kelly Kreisler, Elizabeth Montgomery Collins Jan 2025

Qualitative And Quantitative Analysis Of A Novel Dental Health Education Program To Improve Dental Care Utilization By Bhutanese-, Burmese-, And Swahili-Speaking Refugees In A Midwestern United States City, Chandra Swanson, Katherine Gordon, Kelly Kreisler, Elizabeth Montgomery Collins

2020-Current year OA Pubs

No abstract provided.


System Readiness And The Patient Care Pathway For Alzheimer's Disease Diagnosis And Treatment, B Joy Snider, Et Al. Jan 2025

System Readiness And The Patient Care Pathway For Alzheimer's Disease Diagnosis And Treatment, B Joy Snider, Et Al.

2020-Current year OA Pubs

Promising therapeutic interventions that target the underlying pathophysiology are changing the landscape of Alzheimer's disease (AD) research. The AD care pathway must be transformed to meet the challenge of bringing these new therapies to the increasing number of people living with AD within the existing healthcare framework. Challenges include identifying patients who may benefit from treatment interventions early in the course of the disease, ensuring that diagnostic tools are accessible and accurate, and developing capabilities to monitor the effectiveness of interventions over time. These challenges must be addressed at all levels, from primary care settings to tertiary treatment centers; this …


Building Toward A Standard Model For Perinatal Mental Healthcare, R Christopher Sheldrick, Megan Deichen Hansen, Nicole H Cirino, Mary Kimmel, Courtney King, Deirdre Logan, Rena Menke, Karen Tabb Jan 2025

Building Toward A Standard Model For Perinatal Mental Healthcare, R Christopher Sheldrick, Megan Deichen Hansen, Nicole H Cirino, Mary Kimmel, Courtney King, Deirdre Logan, Rena Menke, Karen Tabb

2020-Current year OA Pubs

BACKGROUND: Healthcare decision-making relies on models that synthesize complex components such as disease epidemiology, diagnostic accuracy, and treatment efficacy. A healthcare model serves as a framework to integrate health systems research, biological understanding, and diverse perspectives on health, enabling decision-makers to optimize access, quality, cost, and equity. The National Academy of Sciences, Engineering, and Medicine (NASEM) has underscored the need for a shared conceptualization in behavioral sciences to unify definitions and facilitate data synthesis. To achieve this, a standard model of perinatal mental healthcare is imperative.

OBJECTIVE: We propose the development of a standard model of perinatal mental healthcare, analogous …


Bronchus-Associated Lymphoid Tissue In Lung Transplantation: A Facilitator Of Rejection Or Regulator Of Tolerance?, Alexander N Wein, Charles R Liu, Daniel Kreisel Jan 2025

Bronchus-Associated Lymphoid Tissue In Lung Transplantation: A Facilitator Of Rejection Or Regulator Of Tolerance?, Alexander N Wein, Charles R Liu, Daniel Kreisel

2020-Current year OA Pubs

The role of bronchus-associated lymphoid tissue (BALT) in the regulation of immune responses to transplanted lungs remains an area of interest and controversy. Early studies in a rat pulmonary transplant model suggested BALT may accelerate rejection of grafts by inducing a local and systemic inflammatory response. Such observations were corroborated in intrapulmonary tracheal transplant models in the rat. While some human studies have described the presence of BALT in grafts that have been chronically rejected, others did not observe an association between induction of BALT and adverse outcomes. More recent investigations have found that BALT, enriched in immunoregulatory cell populations, …


Adapting Data Science Competencies By Role And Purpose: Voice Ai, David A Dorr, Andrea Krussel, Rachel Hauck, Christie Jackson, Abhijeet Dalal, Steven Bedrick, Philip R O Payne, Bridge2ai-Voice Consortium;, William Hersh Jan 2025

Adapting Data Science Competencies By Role And Purpose: Voice Ai, David A Dorr, Andrea Krussel, Rachel Hauck, Christie Jackson, Abhijeet Dalal, Steven Bedrick, Philip R O Payne, Bridge2ai-Voice Consortium;, William Hersh

2020-Current year OA Pubs

Competencies help define the skills and knowledge needed by learners. Often broad, educators integrate competencies to provide a framework for curricula or professional standards. For data science, the rate of change in the field, role variations, and specificity in key applications can be challenging. Our objective was to adapt general data science competencies for different learner roles in an emerging area: the clinical utility of Voice, Language, and Speech-based Artificial Intelligence/Machine Learning (AI/ML). Using a persona-inductive approach, we adapted competencies to support learners from varying professional and educational backgrounds and implemented these adaptations in a multi-institutional summer school. Results from …


No Evidence Of Mmr Induced Trained Immunity To Prevent Sars Cov2: Results From A Multi-Centre Rct, Sinead Delany-Moretlwe, George Kyei, Erik Dubberke, Linda Yun, Leon Du Toit, Mary Politi, Ben Swan, Anne Deschryver, Sherry Mckinnon, Ananya Gupta, Shabaana Khader, Michael S Avidan, Et Al. Jan 2025

No Evidence Of Mmr Induced Trained Immunity To Prevent Sars Cov2: Results From A Multi-Centre Rct, Sinead Delany-Moretlwe, George Kyei, Erik Dubberke, Linda Yun, Leon Du Toit, Mary Politi, Ben Swan, Anne Deschryver, Sherry Mckinnon, Ananya Gupta, Shabaana Khader, Michael S Avidan, Et Al.

2020-Current year OA Pubs

BACKGROUND: Measles-containing vaccines (MCV), by training innate immune cells, are hypothesized to prevent severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection and coronavirus disease 2019 (COVID-19).

METHODS: In this international, double-blind, placebo-controlled trial, we randomly assigned adults, 18 years and older, to receive MCV or saline. The primary outcome was polymerase chain reaction (PCR) confirmed symptomatic COVID-19, up to 60 days after intervention. Secondary outcomes were PCR-confirmed symptomatic COVID-19 and serologically confirmed SARS-CoV-2 infection, up to 150 days after intervention.

RESULTS: Of 3411 randomised participants, the modified intention-to-treat population included 1607 in the MCV and 1545 in the saline group. …


Parp Inhibitors In Melanoma Treatment: Potential, Challenges, And Future Directions, Angela Anaeme, Karen Moussa, Abdallah N Mansour, George Nassief, George Ansstas Jan 2025

Parp Inhibitors In Melanoma Treatment: Potential, Challenges, And Future Directions, Angela Anaeme, Karen Moussa, Abdallah N Mansour, George Nassief, George Ansstas

2020-Current year OA Pubs

Genome integrity is a critical driver of cellular stability, and defects in the processes that maintain genome health are potent sources of cancer progression. Homologous recombination deficiency (HRD), which damages cells through absent or erroneous repair of double-stranded DNA breaks, is a prime example of such cellular dysfunction. Poly(ADP-ribose) polymerase (PARP) inhibitors exploit these aberrancies in the cellular repair process by arresting the ability of the PARP enzyme to repair cellular and genetic damage, inducing the accumulation of DNA damage and cancer cell death. While the utility of PARP inhibitors has been established in many HRD-associated tumors - particularly breast, …


Comparative Effectiveness Of Epilepsy Surgery Versus Additional Anti-Seizure Medications For Lennox-Gastaut Syndrome: Study Protocol For A Multicenter, Mixed-Methods Study, Sandi Lam, Liu Lin Thio, Et Al. Jan 2025

Comparative Effectiveness Of Epilepsy Surgery Versus Additional Anti-Seizure Medications For Lennox-Gastaut Syndrome: Study Protocol For A Multicenter, Mixed-Methods Study, Sandi Lam, Liu Lin Thio, Et Al.

2020-Current year OA Pubs

INTRODUCTION: Lennox-Gastaut Syndrome (LGS) is a severe developmental epileptic encephalopathy without a known cure. Management of symptoms requires substantial care. Treatment options include anti-seizure medications, dietary therapy, and epilepsy surgery. Two main treatment pathways for patients with LGS with drug resistant epilepsy are additional anti-seizure medications or epilepsy surgery, which have been reported to be effective in reduction of seizure burden and improving quality of life. No studies have directly compared the outcomes of using epilepsy surgery versus using additional anti-seizure medications for the treatment of LGS.

METHODS: This study is a multicenter, mixed-methods comparative effectiveness study of LGS patients …


"I Feel Like People Would Look At Me Like I'M Dirty Or Like I'M A Thief:" A Qualitative Exploration Of Interpersonal And Intrapersonal Experiences That Influence Recovery Among Adults At Risk Of Opioid-Related Overdose, Hannah S Szlyk, Sara Jones, Jordan Michener, Zhuoran Zhang, Nina Kaiser, Heidi Holtz, Melody Rachel Konadu Frempong, Husain Lateef, William Hutson, Patricia Cavazos-Rehg Jan 2025

"I Feel Like People Would Look At Me Like I'M Dirty Or Like I'M A Thief:" A Qualitative Exploration Of Interpersonal And Intrapersonal Experiences That Influence Recovery Among Adults At Risk Of Opioid-Related Overdose, Hannah S Szlyk, Sara Jones, Jordan Michener, Zhuoran Zhang, Nina Kaiser, Heidi Holtz, Melody Rachel Konadu Frempong, Husain Lateef, William Hutson, Patricia Cavazos-Rehg

2020-Current year OA Pubs

OBJECTIVE: Due to the nature of early termination of treatment and the transient lifestyles of many people who use substances, many providers are unaware of the factors that may have contributed to their client's engagement in treatment. Treatment providers and researchers need a better understanding of intrapersonal and interpersonal factors that impact recovery from the clients' perspective. This study uses qualitative methods to explore these factors.

METHODS: Eligible participants were adults who had used opioids and/or stimulants and were receiving treatment at a consenting facility. Participants completed one-month follow-up interviews after using a mobile health intervention. Transcripts were analyzed using …


Upper Limb Motor Recovery In Chronic Stroke-Longitudinal Aggregate Analysis From Control Group Outcomes, Fabien Scalzo, Robert A Coker, Lauren Souders, Leo Petrossian, Kern Bhugra, Lauren Sheehan, Eric C Leuthardt, Alexander R Carter Jan 2025

Upper Limb Motor Recovery In Chronic Stroke-Longitudinal Aggregate Analysis From Control Group Outcomes, Fabien Scalzo, Robert A Coker, Lauren Souders, Leo Petrossian, Kern Bhugra, Lauren Sheehan, Eric C Leuthardt, Alexander R Carter

2020-Current year OA Pubs

INTRODUCTION: This study examines the effects of regular physical activity on upper extremity motor recovery during the late subacute and chronic phases of stroke.

METHODS: Data were aggregated from 20 studies comprising 368 participants in control groups receiving usual care or general rehabilitation without specialized interventions. To isolate the impact of non-specific physical activity, studies involving robotics or task-specific therapies were excluded.

RESULTS: The primary outcome was the change in Fugl-Meyer Assessment of Upper Extremity (FMA-UE) motor scale. The pooled effect size for FMA-UE change was small and non-significant (Cohen's

DISCUSSION: These findings provide a quantitative benchmark for expected gains …


Barriers And Facilitators To Implementing A Task-Sharing Mental Health Intervention For Sickle Cell Disease Populations In Low- And Middle-Income Countries: A Qualitative Analysis Using The Consolidated Framework For Implementation Research (Cfir), John Patena, Leah Elster, Tania Hameed, Sumedha Kulkarni, Alden Yuanhong Lai, Annika C Sweetland, Joyce Gyamfi, Temitope Ojo, Angela Odoms-Young, Charmaine Royal, Emmanuel Peprah Jan 2025

Barriers And Facilitators To Implementing A Task-Sharing Mental Health Intervention For Sickle Cell Disease Populations In Low- And Middle-Income Countries: A Qualitative Analysis Using The Consolidated Framework For Implementation Research (Cfir), John Patena, Leah Elster, Tania Hameed, Sumedha Kulkarni, Alden Yuanhong Lai, Annika C Sweetland, Joyce Gyamfi, Temitope Ojo, Angela Odoms-Young, Charmaine Royal, Emmanuel Peprah

2020-Current year OA Pubs

BACKGROUND: People living with Sickle Cell Disease (SCD) experience higher rates of common mental disorders (CMD). There is an alarming treatment gap in the provision of adequate mental health services for CMDs in low- and middle-income countries (LMIC). One solution is the implementation of task-sharing interventions such as the Friendship Bench which utilizes concepts of problem-solving therapy (PST). This investigation uses a qualitative study design to evaluate the acceptability and feasibility of implementing a PST-based task-sharing mental health intervention for SCD populations in LMICs using the Consolidated Framework for Implementation Research (CFIR).

METHODS: Purposive, convenience, and snowball sampling strategies were …


Molecular Chaperones At The Host-Virus Interface: Heat Shock Protein Roles In Hiv-1 And Emerging Insights For Hiv-2 And Dual Infection, Sabastine Eugene Arthur, Kirk Klogo, Emmanuel Kobina Mensah, Maame Ama Pentsiwa Cudjoe, Adomia Baaba Mensah, Nyamekye Amoabeng Ankrah, Catherine Laaripuoh Omosule, Evelyn Yayra Bonney, George Boateng Kyei Jan 2025

Molecular Chaperones At The Host-Virus Interface: Heat Shock Protein Roles In Hiv-1 And Emerging Insights For Hiv-2 And Dual Infection, Sabastine Eugene Arthur, Kirk Klogo, Emmanuel Kobina Mensah, Maame Ama Pentsiwa Cudjoe, Adomia Baaba Mensah, Nyamekye Amoabeng Ankrah, Catherine Laaripuoh Omosule, Evelyn Yayra Bonney, George Boateng Kyei

2020-Current year OA Pubs

Heat shock proteins (HSPs) are essential molecular chaperones involved in protein folding, cellular stress responses, and homeostasis. Recent studies reveal their critical and dual roles in the human immunodeficiency virus (HIV) life cycle, both promoting and restricting viral replication, latency, and immune modulation. This review synthesizes current evidence on how key HSP families, HSP70, HSP90, and HSP40, interact with HIV proteins such as Tat, Rev, Nef, and Vpx, affecting viral transcription, protein trafficking, and latency. We also highlight Heat Shock Factor 1 (HSF1) as a direct regulator of the HIV-1 long terminal repeat (LTR), facilitating latency reversal via recruitment of …


The Gut Microbiome And Gastrointestinal Cancers: Mechanisms, Biomarkers And Therapeutic Opportunities, Sarbjeet Kaur Makkar, Kumar S Bishnupuri Jan 2025

The Gut Microbiome And Gastrointestinal Cancers: Mechanisms, Biomarkers And Therapeutic Opportunities, Sarbjeet Kaur Makkar, Kumar S Bishnupuri

2020-Current year OA Pubs

Gastrointestinal (GI) cancers remain a leading global cause of cancer-related mortality, significantly impacting public health and healthcare systems worldwide. Emerging evidence underscores the critical role of gut microbiome dysbiosis-characterized by disrupted microbial diversity and function-in GI carcinogenesis. Utilizing recent advancements in multi-omics technologies and sophisticated computational biology, researchers have elucidated distinct microbial signatures associated with colorectal, gastric, hepatobiliary, pancreatic, and esophageal cancers. This review comprehensively analyzes the primary mechanisms through which gut microbes contribute to cancer development and progression, encompassing genotoxicity, chronic inflammation, metabolic dysregulation, epigenetic modifications, and immunomodulation. Moreover, we explore innovative microbiome-derived biomarkers for potential clinical applications, including …


The Role Of Dorsal Raphe Nucleus Neuropeptides In Reward And Aversion, Kathryn Braden, Daniel C Castro Jan 2025

The Role Of Dorsal Raphe Nucleus Neuropeptides In Reward And Aversion, Kathryn Braden, Daniel C Castro

2020-Current year OA Pubs

The dorsal raphe nucleus is a critical node for affective and motivated circuits in the brain. Though typically known as a serotonergic hub, the dorsal raphe nucleus is also highly enriched in a variety of neuropeptides. Recent advances in biotechnology and behavioral modeling have led to a resurgence in neuropeptide research, allowing investigators to target unique peptide systems with unprecedented clarity. Here, we review and discuss multiple neuropeptide systems in dorsal raphe and consider how their activity may contribute to reward and aversion. While this is not an exhaustive review, this short overview will highlight the many opportunities available to …


The Perfect Storm: Unraveling The Interplay Of Genetic Predisposition And Viral Triggers In Type 1 Diabetes Pathogenesis, Daniel A Veronese-Paniagua, Jeffrey R Millman Jan 2025

The Perfect Storm: Unraveling The Interplay Of Genetic Predisposition And Viral Triggers In Type 1 Diabetes Pathogenesis, Daniel A Veronese-Paniagua, Jeffrey R Millman

2020-Current year OA Pubs

Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the T cell-mediated destruction of insulin-secreting pancreatic β cells, leaving patients reliant on exogenous insulin to establish normoglycemia. Despite advancements in glucose management, the precise pathological mechanisms linking genetic predisposition and environmental triggers to the loss of immune tolerance remain incompletely understood, hindering the development of preventative therapies. This comprehensive review synthesizes clinical, experimental, and epidemiological data to detail the intricate pathogenesis of T1D, focusing on the convergence of autoimmunity, high-risk genetics, and enteroviral infection. We discuss how T1D is fundamentally a disease caused by failures in central and …