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Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study
Background: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence of functional dystrophin protein. Viltolarsen, an exon 53 skipping therapy, significantly increased dystrophin levels in patients with DMD. Presented here are completed study results of \u3e 4 years of functional outcomes in viltolarsen-treated patients compared to a historical control group (Cooperative International Neuromuscular Research Group Duchenne Natural History Study [CINRG DNHS]). Objective: To evaluate the efficacy and safety of viltolarsen for an additional 192 weeks in boys with DMD. Methods: This phase 2, open-label, 192-week long-term extension (LTE) study (NCT03167255) evaluated the efficacy and safety of viltolarsen in participants aged 4 to \u3c 10 years at baseline with DMD amenable to exon 53 skipping. All 16 participants from the initial 24-week study enrolled into this LTE. Timed function tests were compared to the CINRG DNHS group. All participants received glucocorticoid treatment. The primary efficacy outcome was time to stand from supine (TTSTAND). Secondary efficacy outcomes included additional timed function tests. Safety was continuously assessed. Results: For the primary efficacy outcome (TTSTAND), viltolarsen-treated patients showed stabilization of motor function over the first two years and significant slowing of disease progression over the following two years compared with the CINRG DNHS control group which declined. Viltolarsen was well tolerated, with most reported treatment-emergent adverse events being mild or moderate. No participants discontinued drug during the study. Conclusions: Based on the results of this 4-year LTE, viltolarsen can be an important treatment strategy for DMD patients amenable to exon 53 skipping
Effects of Acute and Repeated Alcohol Exposure on Expression of Synaptic-Associated Genes in the Male and Female Mouse mPFC
Alcohol use disorder (AUD) is a serious chronic brain disease; in 2021 there were ~29.5 million people in the U.S. diagnosed with AUD. Individuals with AUD often show cognitive impairment such as risky decision-making, difficulties with impulse control, and working memory deficits. This impairment is associated with structural and functional changes in the prefrontal cortex (PFC), an extensively interconnected region of the frontal lobe involved in executive control of goal-directed behaviors. Chronic alcohol exposure in rodents has been seen to cause deficits in performance in behavioral tasks which assess mPFC function such as working memory and behavioral flexibility. It is thought that the remodeling of mPFC synapses contributes to alcoholrelated cognitive impairment, but it is not known which specific pathways and genes are responsible for alcohol\u27s long-term effects. Given that synaptic transmission is likely an important contributor to the development of these long-term cognitive deficits, it is surprising that relatively little is known about how alcohol affects expression of the synaptic vesicle fusion machinery in males and females. Thus, the purpose of this study was to investigate how acute and repeated alcohol exposure modify the gene expression of synaptic vesicle-associated proteins, including HSF1, VAMP1, VAMP2, and Syt1 in the mPFC of male and female mice Through qPCR, we found significant sex and alcohol exposure differences in synaptic gene expression. In males, Vamp1 expression increased after acute alcohol exposure and decreased after repeated alcohol exposure. In females, Vamp2 expression increased after acute and repeated ethanol exposure and Hsf1 expression increased after repeated ethanol exposure. These findings suggest that ethanol differentially induces changes in the expression of synaptic genes in male and female mPFC, which likely impacts neurotransmission through changes in the synaptic vesicle fusion machinery. Additionally, this suggests that there is a compensatory mechanism to counteract the effects of multiple ethanol exposures, which is present in the male mPFC but not in the female mPFC. Therefore, future studies should investigate potential sex differences through a systems biology approach by investigating how alcohol can regulate expression and function of postsynaptic GABA and glutamate receptors, as well as GABA and glutamate release
Navigating Systemic Limitations on Public Defense
Under both the United States Constitution and the rulings of the U.S. Supreme Court, public defender offices are tasked with effectively defending their clients’ human rights to life, liberty, property, and to the due process of law. In the past, public defender offices across New York State have faced barriers in providing such representation, as they have historically suffered from a lack of resources and funding. More recently, however, their problems have shifted to a lack of accessibility of programs for their indigent clients. In our research, we will identify the systemic limitations on public defense that the Broome County Public Defender’s Office encounters and evaluate how they navigate those limitations. We will be conducting a review of the legal, financial, and other external restrictions that have been placed on the office, and consider the effectiveness of the legal practices of triage and holistic defense in combating these limitations.https://orb.binghamton.edu/research_days_posters_2023/1086/thumbnail.jp
Examination of Hepatic Cancer Rates in Varying Trichloroethylene Concentrations in the Contamination Zone of the IBM-Endicott Chemical Spill
In 1979, International Business Machines spilled 3700 gallons of a volatile organic compound, trichloroethylene (TCE), beneath its facility in Endicott, NY. TCE is a known carcinogen and has been associated with elevated levels of hepatic cancer in previous toxicological studies. For these reasons, the researchers conducted a case-control study to determine if there was an elevated risk of hepatic cancer in Endicott preceding the spill. In addition to common practices employed in case-control studies, such as survey data collection, the researchers also employed multidisciplinary methods of data visualization to illustrate the relationship between the spill location and elevated hepatic cancer rates. The geographic relationship between the number of hepatic cancer cases and their locations relative to groundwater TCE concentrations indicates that there is a strong correlation between the presence of TCE and an increased incidence of hepatic cancer. This conclusion is further supported by cancer registry data, which depicts Endicott as having a higher incidence of hepatic cancer than Broome County and New York State.https://orb.binghamton.edu/research_days_posters_2023/1089/thumbnail.jp
Misinformation: The Effects on Past and Future Outbreaks
Misinformation is a major challenge in addressing large-scale acute infectious outbreaks and has been shown to encourage vaccine hesitancy and decrease vaccination rates. This study examines the effects of misinformation on vaccine hesitancy by comparing studies from past and present outbreaks. Specifically, I investigate the following research question: What are the effects of misinformation on vaccine hesitancy during past and current outbreaks and what are effective preventative measures? I analyze a range of studies using a systematic review approach to identify similarities and differences in the spread of misinformation across outbreaks. My findings shed light on how and why misinformation spreads and the effects it has on combating disease. The implications of my study are significant, as the World Health Organization has ranked misinformation as one of the top ten threats to global health. By identifying patterns and potential solutions, my study contributes to the larger conversation about addressing misinformation and vaccine hesitancy.https://orb.binghamton.edu/research_days_posters_2023/1001/thumbnail.jp
Prosperity Gospel
Mirroring the capitalist world around it, the American Protestant Church preaches a prosperity gospel, a theological, economic, and social force within American religion. This research examines the history and emergence of the prosperity gospel and its relationship to capitalism. Its theology teaches adherents that they will be blessed with material prosperity in the form of health and wealth if they practice its teachings, that is, if they are capitalists. The core idea that religion begets wealth bridges Christianity and capitalism, allowing believers to believe they are Christians while unconditionally accepting market capitalism. In this research, I demonstrate how three streams of thought led nominations of American Protestantism to this point: New Thought, Pentecostalism, and American gospel; in the first, believers accessed supernatural powers of healing through prayer; the second followed the ideas of New Thought but emphasized material prosperity over health; and, finally, these elements coalesced with American individualism and the so-called Protestant work-ethic (of Max Weber) into the prosperity gospel\u27s message that this capitalism masquerading as Christianity would bring blessings upon followers.https://orb.binghamton.edu/research_days_posters_2023/1018/thumbnail.jp
Effect of Alcohol on Nutritional Habits and Mental Well-Being
Alcoholism is a pervasive disease believed to affect nutritional choices and mental state. This study examined the effects of drinking on diet quality and mood. An anonymous online survey was administered through a Google survey that was distributed on social media platforms. The survey included basic demographic questions and questions aimed at gauging alcohol consumption, nutritional habits, and self perceived mental well-being. Pearson Correlation Coefficient was used to analyze data in SPSS version 25.0. There is an association between binge drinking and depressed mental health. In addition, alcohol drinking influences food choices and overall mood after a night of drinking. Future research can further explore the relationship between dietary choices, amount of alcohol, and mental statushttps://orb.binghamton.edu/research_days_posters_2023/1053/thumbnail.jp
Relationship Between Binge Drinking, Food Restriction, and Mental Health in College Students
Binge drinking is a prevalent problem among college students and it is impacting many aspects of lives. This study focuses on the detriments of binge drinking on college students’ nutrition and mental health. An anonymous online survey was administered through Facebook, Instagram, Snapchat, and Discord. The survey included basic demographic questions and questions on frequency of drinking, eating habits while drinking, typical daily food consumption, and mental-well being. Pearson Correlation Coefficient was used to analyze data in SPSS version 25.0. There is an association between binge drinking, food choices, and mental health among college students. Because of the effects of binge drinking, it is important to be conscious of drinking habits to protect physical and mental health.https://orb.binghamton.edu/research_days_posters_2023/1063/thumbnail.jp
The Influence of Colored Light on Mexican Jumping Bean Larva (Laspeyresia saltitans) Activity
The Mexican jumping bean larva, Laspeyresia saltitans, is a moth larva that lives in the seeds of a fern, Sebastiana pavoniana, found throughout deserts in Mexico. Mexican jumping bean larvae are known to jump to avoid the intense heat and sunlight of their environment, but little is known about the specific cues that induce this behavior. We analyzed how different wavelengths of light affected the activity of the jumping bean larvae through exposure to red, green, purple and white light. Larval movement from their original position was measured, and we determined the greatest displacement occurred under red light, and the least activity under purple light, highlighting a mechanism to explain locomotion. As Mexican jumping beans and other insects are exposed to different wavelengths of light throughout the day in their natural habitat, this study helps provide insight on how the composition of sunlight may affect insect activity and behavior.https://orb.binghamton.edu/research_days_posters_2023/1095/thumbnail.jp
State-Sponsored Disinformation about Uyghurs: Euphemism as Genocide Denial
My research uncovers the use of euphemism by the Chinese government to spread false narratives and deny allegations of human rights abuses and genocidal acts against the Uyghur ethnic minority. Euphemistic names for policies like vocational training centers and poverty alleviation are rooted in information campaigns that deceitfully frame Chinese actions as positive and beneficent. Likewise, the Chinese government labels Uyghurs as terrorists, harmfully and falsely characterizing an entire ethnicity as criminal. My analysis contributes to the field of Disinformation Studies by highlighting the specific function of the device, euphemism, as an intentionally false distortion of narrative that causes significant harm while also bridging the gap between the study of genocide and the disinformation narratives that falsely justify it.https://orb.binghamton.edu/research_days_posters_2023/1098/thumbnail.jp