Medicine and Health Sciences Commons^™

First Successful Ovarian Cortex Allotransplant To A Turner Syndrome Patient Requiring Immunosuppression: Wide Implications, Sherman J Silber, Sierra Goldsmith, Benjamin Rubinoff, Eduardo Kelly, Rowena Delos Santos, Anibal Melo, Daniel Brennan

2020-Current year OA Pubs

OBJECTIVE: To determine whether we can safely and successfully transplant an ovary tissue allograft from a nonidentical donor to her Turner syndrome sister.

DESIGN: Transplantation of cryopreserved ovary tissue, as well as fresh transplantation of ovarian tissue between identical twins, is now well established with numerous reported successful cases. However, there have not yet been any ovary transplants between nonidentical women requiring immunosuppression (ovary allotransplant). This could be a much more common indication for ovary tissue transplantation if safe and reliable immunosuppression were available.

PATIENT(S): A 20-year-old amenorrheic woman with nonmosaic 45-XO Turner syndrome requested ovary tissue transplantation from her …

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 …

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 …

Depression And Weight Loss Trajectories During An Integrated Behavioral Intervention: Within-Treatment Analysis Of The Rainbow Trial, Bailey W. Osweiler, Daphne Lew, Nan Lv, Jun Ma, Thomas G. Kannampallil

2020-Current year OA Pubs

INTRODUCTION: Depression and obesity are two of the most common chronic conditions among US adults. The Research Aimed at Improving Both Mood and Weight (RAINBOW) study was the first randomized clinical trial to demonstrate the effectiveness of an integrated behavioral treatment for depression and obesity. We aimed to characterize the trajectories of both depression symptoms and weight loss during the RAINBOW intervention.

METHODS: Participants (N = 201) randomly assigned to the RAINBOW treatment arm who provided serial data on depression symptoms (via the 9-item Patient Health Questionnaire [PHQ9]) and self-measured weight were included. Group-Based Multi-Trajectory Modeling is a statistical technique …

Vagus Nerve Stimulation In Treatment-Resistant Depression: A One-Year, Randomized, Sham-Controlled Trial, Charles R Conway, Christopher L Kriedt, Et Al.

2020-Current year OA Pubs

BACKGROUND: Few treatments are available for individuals with marked treatment-resistant depression (TRD).

OBJECTIVE: Evaluate the safety and effectiveness of FDA-approved adjunctive vagus nerve stimulation (VNS) in patients with marked TRD.

METHODS: This 12-month, multicenter, double-blind, sham-controlled trial included 493 adults with marked treatment-resistant major depression who were randomized to active or no-stimulation sham VNS for 12 months. The primary outcome was percent time in response across months 3-12, with response defined as a ≥50 % change from baseline on the Montgomery-Åsberg Depression Rating Scale (MADRS). Several secondary endpoints were evaluated.

RESULTS: Overall, 88.4 % of participants completed the trial. Percent …

Exploring Factors Contributing To Patient Decision-Making In The Care Journey To Elective Hernia Care In Kenya, Helen W. Li, Jesse Kiprono Too, Sarah Nyanchama Nyariki, Charles Nathan Nessle, Sara Malone, Rachel Matsumoto, Teddy Ashibende Aurah, Jeffrey A. Blatnik, Joanna Hunter-Squires, Ivan Seno Saruni

2020-Current year OA Pubs

BACKGROUND: Capacity for elective general surgical care is an important reflection of a health system's ability to meet a population's surgical needs and is currently known to be inadequate in many low- and middle-income countries. Patient agency is a key, understudied factor which shapes how and when patients ultimately decide to engage with formal care. Understanding factors which influence patient care seeking activity can have important implications for how current and future health systems may be utilized. This study aims to explore how patients approach the navigation and triage of their elective hernia condition within the Kenyan surgical care system. …

Cervical Cancer In Women With Hiv: A Call To Action For Equitable Prevention In Low- And Middle-Income Countries, Jonathan Klutse, Yeena Abla Tay, Dzidzor Aku Attoh, Emmanuel Frimpong Gyekye, Charlotte Borteley Bortey, Esenam Dzifa Buatsi, Charlayne Cherylyn Oppong, Helena Lamptey, Collins Stephen Ahorlu, George Boateng Kyei, Evelyn Yayra Bonney

2020-Current year OA Pubs

Cervical cancer is preventable; however, it remains the leading cause of death in low- and middle-income countries (LMICs). Women with HIV (WWHs) have a sixfold higher risk of developing and dying from cervical cancer than women without HIV. Cervical cancer can be prevented by vaccination against high-risk human papillomaviruses (hrHPVs) and by screening for and treating precancer cervical lesions. While these preventive measures are routinely available to WWHs in developed countries, they are lacking in most LMICs, where the burden of HIV and cervical cancer is the highest. To prevent cervical cancer deaths among WWHs in LMICs, it is imperative …

Usability Of The Mayo Test Drive Remote Self-Administered Web-Based Cognitive Screening Battery In Adults Aged 35-100 With And Without Cognitive Impairment, Jay S Patel, Jason Hassenstab, Et Al.

2020-Current year OA Pubs

BACKGROUND: Mayo Test Drive (MTD): Mayo Test Development through Rapid Iteration, Validation and Expansion, is a web-based remote cognitive assessment platform for self-administered neuropsychological measures with previously demonstrated validity and reliability. We examined the usability of MTD and hypothesized that completion rates would be greater than 90%. We explored whether completion and participation rates differed by cognitive status and demographic factors.

METHODS: A total of 1,950 Mayo Clinic Study of Aging and Mayo Alzheimer's Disease Research Center participants (97% White, 99% Non-Hispanic) were invited to participate in this ancillary, uncompensated remote study. Most invitees were cognitively unimpaired (CU;

RESULTS: Baseline …

An Exploratory Graphical Analysis Of The Montgomery-Åsberg Depression Rating Scale Pre- And Post-Treatment Using Pooled Antidepressant Trial Secondary Data, David Byrne, Arunangshu Ghoshal, Fiona Boland, Susan Brannick, Robert M Carney, Pim Cuijpers, Alexandra L Dima, Kenneth E Freedland, Suzanne Guerin, David Hevey, Bishember Kathuria, Vincent Mcdarby, Emma Wallace, Frank Doyle

2020-Current year OA Pubs

BACKGROUND: The 10-item Montgomery-Åsberg Depression Rating Scale (MADRS) is a commonly used measure of depression in antidepressant clinical trials. Numerous studies have adopted classical test theory perspectives to assess the psychometric properties of this scale, finding generally positive results. However, its network configural structure and stability is unexplored across different time-points and treatment groups.

AIMS: To assess the network structure and stability of the MADRS in clinical settings pre- and post-treatment, and to determine a configurally invariant and stable model across time-points and treatment groups (placebo and intervention).

METHOD: Individual participant data for 6440 participants from 14 clinical trials of …

Disclosing Disabilities: Barriers For Medical School Applicants, Sarah Leah Solomon, Aitan E. Magence, Harry Haran, Fern R. Juster, Kristina H. Petersen

2020-Current year OA Pubs

This study investigated the availability of accommodation request procedures for medical school admissions interviews and the accessibility of Technical Standards (TS) for candidates with disabilities (CWD) across Liaison Committee on Medical Education (LCME)-accredited institutions in the United States and Canada. Utilizing a cross-sectional study methodology, surveys were distributed to Deans of Admissions and Disabilities Resource Professionals (DRP) at all LCME-accredited US and Canadian MD programs. Surveys gathered data about interview formats, interview accommodation procedures, and TS accessibility during the 2018-2019 academic year. We received responses from 71 institutions (41.3%), with 56.3% survey completion rate (n = 40). Among respondents, interview …

Efficacy And Safety Of Dostarlimab In Combination With Chemotherapy In Patients With Dmmr/Msi-H Primary Advanced Or Recurrent Endometrial Cancer In A Phase 3, Randomized, Placebo-Controlled Trial (Engot-En6-Nsgo/Gog-3031/Ruby), Matthew A Powell, Carolyn Mccourt, Et Al.

2020-Current year OA Pubs

OBJECTIVES: Part 1 of the RUBY trial (NCT03981796) demonstrated improved survival in patients with primary advanced or recurrent endometrial cancer (EC) treated with dostarlimab plus carboplatin-paclitaxel versus placebo plus carboplatin-paclitaxel. Here, we examine additional efficacy and safety data from patients with mismatch repair deficient/microsatellite instability-high (dMMR/MSI-H) EC in the RUBY trial.

METHODS: Patients were randomized 1:1 to dostarlimab 500 mg or placebo plus carboplatin-paclitaxel every 3 weeks for 6 cycles followed by dostarlimab or placebo every 6 weeks for up to 3 years. In the dMMR/MSI-H population of RUBY Part 1, analysis of progression-free survival by investigator assessment compared with …

Viral Kinetics Among Persons Living With Hiv (Plwh) On Dolutegravir-Based Antiretroviral Regimen: A Retrospective And Prospective Analysis From Selected Hiv Clinics In Ghana, Samuel Badu Nyarko, Joel Adu Twum, Aikins Sarpong Obeng, Helena Dede Djangba, Oksana Ryabinina, Foster Kyei, George Boateng Kyei, Nicholas Ekow Thomford

2020-Current year OA Pubs

BACKGROUND: Dolutegravir (DTG)-based antiretroviral therapy has demonstrated superior efficacy, tolerability, and durability when compared to other HIV treatment regimens. However, monitoring viral kinetics is critical for determining treatment efficacy and making sound judgments. The purpose of this study was to assess viral kinetics in people living with HIV (PLWH) on DTG-based ART and identify characteristics related to virologic response in the Cape Coast Metropolis, Ghana.

METHODS: Among people living with HIV (PLWH) attending HIV clinics between January 2020 and December 2023, a prospective and retrospective analysis of viral kinetics and clinical data were carried out. Data on viral loads, clinical …

Clinical Trials For Wolfram Syndrome Neurodegeneration: Novel Design, Endpoints, And Analysis Models, Guoqiao Wang, Zhaolong Adrian Li, Ling Chen, Heather Lugar, Tamara Hershey

2020-Current year OA Pubs

OBJECTIVE: Wolfram syndrome, an ultra-rare condition, currently lacks effective treatment options. The rarity of this disease presents significant challenges in conducting clinical trials, particularly in achieving sufficient statistical power (e.g., 80%). The objective of this study is to propose a novel clinical trial design based on real-world data to reduce the sample size required for conducting clinical trials for Wolfram syndrome.

METHODS: We propose a novel clinical trial design with three key features aimed at reducing sample size and improve efficiency: (i) Pooling historical/external controls from a longitudinal observational study conducted by the Washington University Wolfram Research Clinic. (ii) Utilizing …

Endogenous Self-Peptides Guard Immune Privilege Of The Central Nervous System, Min Woo Kim, Wenqing Gao, Cheryl F Lichti, Xingxing Gu, Taitea Dykstra, Jay Cao, Igor Smirnov, Pavle Boskovic, Denis Kleverov, Andrea F M Salvador, Antoine Drieu, Kyungdeok Kim, Susan Blackburn, Clair Crewe, Maxim N Artyomov, Emil R Unanue, Jonathan Kipnis

2020-Current year OA Pubs

Despite the presence of strategically positioned anatomical barriers designed to protect the central nervous system (CNS), it is not entirely isolated from the immune system

Improving Outcomes Of Single-Sided Deaf Cochlear Implant Users By Reducing Interaural Frequency And Loudness Mismatches Through Device Programming, Laura K Holden, Rosalie M Uchanski, Noël Y Dwyer, Ruth M Reeder, Timothy A Holden, Jill B Firszt

2020-Current year OA Pubs

The study aimed to improve outcomes in Nucleus cochlear implant (CI) recipients with single-sided deafness (SSD) by reducing interaural frequency and loudness mismatches through device programming. In Experiment 1a, a modified frequency allocation table (FAT) was created to better match the tonotopicity of the contralateral ear and reduce interaural frequency mismatch. Twenty experienced SSD-CI users completed localization and speech recognition tests with their everyday FAT. Tests were repeated after 6 weeks' use of the modified FAT. Participants compared both FATs for 2 weeks before being tested again with each. For 10 newly implanted SSD-CI recipients (Experiment 1b), Group A was …

Persistent Advanced Hiv Disease In Rural Kwazulu-Natal, South Africa: Trends, Characteristics, And The Urgent Need For Targeted Interventions, Marcel K. Kitenge, Geoffrey Fatti, Ingrid Eshun-Wilson, Peter S. Nyasulu

2020-Current year OA Pubs

BACKGROUND: Advanced HIV disease (AHD) remains a persistent public health challenge. Data regarding the burden, characteristics and predictors of AHD is scarce specifically for rural settings of sub-Saharan Africa. This study aimed to describe trends in annual CD4 count distribution and to characterise adult persons living with HIV (PLWH) on ART who have AHD in rural KwaZulu-Natal, South Africa.

METHODS: A retrospective cohort design of annual CD4 count distribution was conducted among antiretroviral therapy (ART) patients aged 18 years and older. We used routinely collected data from adults receiving ART in Eshowe and Mbongolwane areas in KwaZulu-Natal, South Africa, between …

Revisiting Abdominal Wall "Morbidity" Of The Extensile Anterolateral Approach To The Thoracolumbar Spine, Alekos A Theologis, Andrew P Collins, Kanwar Parhar, Munish C Gupta

2020-Current year OA Pubs

PURPOSE: To assess patients' perceptions of their abdominal wall following extensile anterolateral approaches to the thoracolumbar spine for adult spinal deformity (ASD) using validated questionnaires.

METHODS: Adults who underwent anterior-posterior thoracolumbar spinal operations to the pelvis for ASD in which the anterior fusion was performed through an extensile anterolateral approach were reviewed. Three questionnaires were administered at least 1 year following surgery and included The Abdominal Core Health Quality Collaborative Survey (AHS-QC), The Patient Scar Assessment Scale (PSAS), and The Anterior Abdominal Incision Questionnaire (AAIQ).

RESULTS: Fifty-one patients (80.4% female, median age 65 years) were included. Average follow-up was 2.8 …

Carpal Tunnel Syndrome And Occupational Co-Exposure To Biomechanical Factors And Neurotoxic Chemicals Using Job-Exposure Matrices And Self-Reported Exposure: Findings From The Constances Cohort, Julie Bodin, Clémence Rapicault, Alexis Descatha, Marc Fadel, Fabien Gilbert, Bradley Evanoff, Nathalie Bonvallot, Marcel Goldberg, Marie Zins, Yves Roquelaure

2020-Current year OA Pubs

OBJECTIVE: To study the association between occupational co-exposure to biomechanical risk factors, potentially neurotoxic chemicals and carpal tunnel syndrome (CTS) in a large cohort of French workers, using two methods to estimate chemical exposure: job-exposure matrices (JEM) and self-reported exposure.

METHODS: A randomly selected sample of adults were included between 2012 and 2018 in the French cohort CONSTANCES. Self-reported CTS was assessed using the first self-administered follow-up questionnaire, sent out approximately one year after baseline. Occupational exposure to biomechanical risk factors was assessed using self-administered questionnaire completed at inclusion. Lifetime occupational exposure to chemicals was assessed using two different methods: …

Surgeon Interpretation Of Patient-Reported Outcome Measures In Upper Extremity Osteoarthritis, Rick S. Looman, Koen Oude Nijhuis, Sina Ramtin, David Ring, Daniel A. London, Ryan Calfee, Niels Brinkman, Science Of Variation Group

2020-Current year OA Pubs

PURPOSE: Measures that quantify levels of pain intensity, incapability, symptoms of distress, and unhelpful thinking were designed as research tools. We studied how surgeons interpret and act on these scores in the care of individual patients.

METHODS: In an online experiment, 67 upper limb specialists from the Science of Variation Group reviewed 10 fictious patient scenarios of trapeziometacarpal, wrist and elbow osteoarthritis (OA) and randomized elements of radiographic severity, levels of discomfort and incapability, deprivation index score (social health), and levels of distress and unhelpful thinking (mental health). Multilevel mixed-effects linear regression identified patient factors associated with surgeon concern, enthusiasm …

Treatment Of Hereditary Hemorrhagic Telangiectasias With Sclerotherapy: A Case Series, Jenny Ji, Andrew M Peterson, Jay F Piccirillo

2020-Current year OA Pubs

BACKGROUND: Hereditary hemorrhagic telangiectasia (HHT) is characterized by abnormal blood vessel formation. One treatment for HHT-related arteriovenous malformations (AVMs) is sclerotherapy, which collapses the blood vessels by irritating the endothelial lining.

METHODS: This case series describes two HHT patients undergoing in-office sodium tetradecyl sulfate sclerotherapy for non-nasal telangiectasias and AVMs. The first patient had AVMs on the red lip while the second patient had an AVM and several telangiectasias on the tongue.

RESULTS: Both patients' AVMs required only one treatment visit and were no longer noticeable within a week. Neither of the patients had any side effects from treatment.

CONCLUSION: …

Nutcracker Syndrome (A Delphi Consensus), Floor Heilijgers, Suresh Vedantham, Et Al.

2020-Current year OA Pubs

BACKGROUND: Nutcracker syndrome (NCS) describes the symptomatic compression of the left renal vein between the aorta and superior mesenteric artery. Whereas asymptomatic compression is a common radiological finding, patients with NCS can report a range of symptoms. There are no specific diagnostic criteria and interventions include a range of open surgical and endovascular procedures. Therefore, we wished to develop an international consensus document covering aspects of diagnosis, management, and follow-up for patients with NCS.

METHODS: A three-stage modified Delphi consensus was performed. A steering committee developed 37 statements covering 3 categories for patients with NCS: diagnosis, management, and follow-up. These …

Acquired Ductopenia: An Insight Into Imaging Findings, Rachita Khot, Nathan R Shelman, Daniel R Ludwig, Rashmi T Nair, Mark A Anderson, Sudhakar K Venkatesh, Raj Mohan Paspulati, Rex A Parker, Christine O Menias

2020-Current year OA Pubs

Hepatic ductopenia is a pathologic diagnosis characterized by a decrease in the number of intrahepatic bile ducts as a consequence of various underlying etiologies. Some etiologies, such as primary sclerosing cholangitis, primary biliary cholangitis, and ischemic cholangitis, often have distinctive imaging findings. In contrast, other causes such as chronic rejection following liver transplantation, drug-induced biliary injury, infection, malignancy such as lymphoma, and graft-versus-host disease may only have ancillary or non-specific imaging findings. Thus, diagnosing ductopenia in conditions with nonspecific imaging findings requires a multidimensional approach, including clinical evaluation, serological testing, imaging, and liver histology to identify the underlying cause. These …

Alternative Approaches For Monitoring And Evaluation Of Lymphatic Filariasis Following Mass Drug Treatment With Ivermectin, Diethylcarbamazine And Albendazole In East New Britain Province, Papua New Guinea, Krufinta Bun, Benedict Mode, Melinda Susapu, Joyceline Salo, Catherine Bjerum, Michael Payne, Daniel Tisch, Makoto Sekihara, Emanuele Giorgi, Gary J. Weil, Peter U. Fischer, Leanne Robinson, Moses Laman, Christopher L. King

2020-Current year OA Pubs

BACKGROUND: WHO recommends two annual rounds of mass drug administration (MDA) with ivermectin, diethylcarbamazine, and albendazole (IDA) for lymphatic filariasis (LF) elimination in treatment naïve areas that are not co-endemic for onchocerciasis such as Papua New Guinea (PNG). Whether two rounds of MDA are necessary or sufficient and the optimal sampling strategies and endpoints for stopping MDA remain undefined.

METHODS AND FINDINGS: Two cross-sectional studies were conducted at baseline (N = 49 clusters or villages) and 12 months after mass drug administration (MDA) with IDA (N = 47 villages) to assess lymphatic filariasis (LF) by circulating filarial antigenemia (CFA) and …

Nanobody Screening And Machine Learning Guided Identification Of Cross-Variant Anti-Sars-Cov-2 Neutralizing Heavy-Chain Only Antibodies, Peter R. Mcilroy, Zhuoming Liu, Sean Whelan, Et Al.

2020-Current year OA Pubs

Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) continues to persist, demonstrating the risks posed by emerging infectious diseases to national security, public health, and the economy. Development of new vaccines and antibodies for emerging viral threats requires substantial resources and time, and traditional development platforms for vaccines and antibodies are often too slow to combat continuously evolving immunological escape variants, reducing their efficacy over time. Previously, we designed a next-generation synthetic humanized nanobody (Nb) phage display library and demonstrated that this library could be used to rapidly identify highly specific and potent neutralizing heavy chain-only antibodies (HCAbs) with prophylactic and …

Patient Stratification By Genetic Risk In Alzheimer's Disease Is Only Effective In The Presence Of Phenotypic Heterogeneity, Jack Euesden, Muhammad Ali, Chloe Robins, Praveen Surendran, Padhraig Gormley, Alzheimer’S Disease Neuroimaging Initiative, David Pulford, Carlos Cruchaga

2020-Current year OA Pubs

Case-only designs in longitudinal cohorts are a valuable resource for identifying disease-relevant genes, pathways, and novel targets influencing disease progression. This is particularly relevant in Alzheimer's disease (AD), where longitudinal cohorts measure disease "progression," defined by rate of cognitive decline. Few of the identified drug targets for AD have been clinically tractable, and phenotypic heterogeneity is an obstacle to both clinical research and basic science. In four cohorts (n = 7241), we performed genome-wide association studies (GWAS) and Mendelian randomization (MR) to discover novel targets associated with progression and assess causal relationships. We tested opportunities for patient stratification by deriving …

Evolution And Recent Radiation Therapy Advancement In Uganda: A Precedent On How To Increase Access To Quality Radiotherapy Services In Low- And Middle-Income Countries, Awusi Kavuma, Solomon Kibudde, Daniel Kanyike, Joseph Kigula-Mugambe, Tianyu Zhao, Hiram Gay, Baozhou Sun, Jackson Orem

2020-Current year OA Pubs

The evolution of radiation therapy in Uganda has been a journey marked by significant milestones and persistent challenges. Since the inception of radiotherapy services in 1988-1989, there has been a concerted effort to enhance cancer treatment services. The early years were characterized by foundational developments, such as the installation of the first teletherapy units, low-dose-rate brachytherapy units, and conventional simulators, and the recognition of radiation oncologists and medical physicist professionals laid the groundwork for radiotherapy treatment modalities. With more support from the International Atomic Energy Agency, the acquisition of dosimetry equipment, treatment planning systems, and additional professional training signaled a …

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

Cancer-Targeted Pro-Theranostic Bi-Metallic Organo-Coordination Nanoparticles, Hengbo Huang, Lei Fang, Janaka Wansapura, Julie L Prior, Brad Manion, Baogang Xu, Cody Hongsermeier, Nisha Gamadia, Nicole Blasi, Rui Tang, Christopher Egbulefu, Monica Shokeen, James D Quirk, Samuel Achilefu

2020-Current year OA Pubs

No abstract provided.

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 …

Measuring Stigma In Pediatric Oncology: A Cross-Sectional Analysis Of Three Global Sites, Lara E Counts, Sara M Malone, Et Al.

2020-Current year OA Pubs

PURPOSE: Stigma contributes to fear and shame, resulting in delays in care-seeking behavior among individuals with cancer. As a social construct, stigma is affected by language, religion, culture, and local norms. This study explored pediatric cancer stigma at the time of diagnosis across diverse settings through the adaptation of two stigma measures.

METHODS: This study was conducted with adolescents and caregivers of children with osteosarcoma and retinoblastoma at three centers in Jordan, Guatemala, and Zimbabwe. The Stigma-related Social Problems (SSP) and the eight-item Stigma Scale for Chronic Illness (SSCI-8) measures were translated into Arabic, Spanish, and Shona and contextually adapted …