{"title":"Vamorolone: revolutionizing Duchenne muscular dystrophy treatment","authors":"Sameen Mukhtar, Muhammad Haris","doi":"10.18203/issn.2454-2156.intjscirep20240983","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240983","url":null,"abstract":"Duchenne muscular dystrophy (DMD) is a severe inherited neuromuscular disorder characterized by a dystrophin gene mutation, leading to progressive muscle weakness and structural degradation. Current management strategies focus on multidisciplinary approaches to mitigate symptoms and enhance quality of life. Conventional glucocorticoids present challenges due to their complex nature and severe side effects. Vamorolone, a first-in-class dissociative steroidal drug recently Food and Drug Administration (FDA) - approved, distinguishes itself through enduring anti-inflammatory effects with reduced safety concerns. Pharmacologically, Vamorolone's mechanism of action, differentiating it from traditional corticosteroids, involves selective glucocorticoid receptor (GR) modulation and mineralocorticoid receptor (MR) antagonism, offering improved safety and tolerability. Notably, its unique Δ9,11 modification prevents adverse receptor interactions, demonstrating superior safety in inhibiting inflammation across various cell types. This article explores Vamorolone's pharmacokinetics, drug interactions, and adverse effects, underscoring its well-tolerated profile with reversible hypothalamic-pituitary-adrenal axis suppression as a notable concern. Comparative studies against prednisone reveal Vamorolone's efficacy in improving muscle strength with minimal side effects, validated through the pivotal Phase IIb VISION-DMD study. Ultimately, Vamorolone has attained a breakthrough status in DMD treatment. Its endorsement by the FDA underscores Vamorolone as a transformative linchpin, heralding a new era in revolutionizing DMD care.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"91 9","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140665197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jeremiah C. Oginyi, Samuel Chukwu, Kelechi U. Paul, Kenneth C. Mkpuma
{"title":"Genetic diversity and stability analysis based on agro-morphological traits among rice genotypes developed through marker-assisted backcrossing","authors":"Jeremiah C. Oginyi, Samuel Chukwu, Kelechi U. Paul, Kenneth C. Mkpuma","doi":"10.18203/issn.2454-2156.intjscirep20240978","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240978","url":null,"abstract":"Background: The genetic diversity and stability analysis based on agro-morphological traits among rice genotypes developed through SSR marker-assisted backcrossing was evaluated at college of agricultural science (CAS), Ebonyi State niversity, Abakaliki, Nigeria. \u0000Methods: In this study, 9 rice accessions sourced from the Iinstitute of tropical agriculture and food security (ITAFoS), universiti Putra, Malaysia and Ebonyi, Nigeria; were evaluated for yield stability performance. The experiments were laid out in randomized complete block design (RCBD) with three replications each for two seasons.\u0000Results: Result of the evaluated rice accessions showed highly marked morphological variability based on the mean performance and were grouped into three main clusters. Cluster I had CP, cluster II contained Line4, Line5, Line6, and Line8, and cluster III had Line1, Line7, Line12, and Putra-1. The first, 3 principal components were identified and retained for having the Eigen-value >1. The first PC had an eigenvalue of (6.30) with 52.5% proportion of which plant height accounted most for this variation with (0.359) whereas panicle length contributed the least (-0.13). For stability analysis, CP had highest values for most of the parameters. High heritability value was recorded for all nine traits which range between 71.04% and 95.06%. The highest heritability value (95.06%) was found in Days 50% heading while grain width had the lowest (71.04%).\u0000Conclusions: It could be concluded that CP, Putral-1, and line7 were the most stable in terms of yield across the two seasons of this study; hence, recommended for parental materials for hybridization.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"13 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140659655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Atypical electroclinical presentation of Landau Kleffner syndrome: a case report of an 8 year old Nigerian child","authors":"Chuks G. Nwala, Omokhuale M. Ejemierele","doi":"10.18203/issn.2454-2156.intjscirep20240717","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240717","url":null,"abstract":"Epileptic seizures account for common neurologic presentations in paediatric neurology units in developing countries. Atypical presentation of syndromic epilepsy may be missed especially sensory, psychic or autonomic manifestations. An 8 year old male Nigerian child presented with recurrent vomiting and delayed motor milestones, regression of speech (acquired aphasia) with unremarkable birth history or any underlying medical or surgical condition. He had delayed gross motor development and continuous bilateral polyspike waves of slow sleep on EEG. Self-limited autonomic epileptic seizure syndrome which was a strong differential of autonomic seizures in early childhood was not suggestive as his EEG findings failed to demonstrate occipital spike wave pattern nor did photic stimulation evoke any epileptiform waves. Child made significant clinical improvement with anticonvulsants, neuro vitamins, and behavioural therapy, evidenced by regain of non-verbal communication, resolution of autonomic symptoms and temper tantrums. A high index of suspicion should be applied in patients with acquired aphasia with subclinical or recurrent autonomic symptoms.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"32 38","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140372648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Need of rural communities to be engaged and empowered for global health and wellness","authors":"S. Chhabra","doi":"10.18203/issn.2454-2156.intjscirep20240716","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240716","url":null,"abstract":"For health and wellness rural communities do many things with whatever they have, use various modalities of actions for prevention, therapy of disorders, maintenance, promotion of health. Also, for everyday life they do various activities which affect their health. Present article is based on observations, information in context of activities by rural people for their health and wellness. Personal observations, experiences were added to information about actions by rural communities for health and wellness. Rural communities use their own wisdom, do many things with whatever they have for living a healthy life. They use various modalities during pregnancy, birth for mother, new born, persons of all ages. Therapies are used for pregnancy care, birth, post birth, other illnesses, injuries and so on. In modern day practice it has become essential to priorities modes of actions for best therapy, best outcome without harmful effects. In addition to various actions for prevention, therapy, maintenance and promotion of health and wellness, communities do other activities which impact their health. This needs sharing for best of global health. Communities use many modalities for therapy, prevention of disorders, rehabilitation and health promotion. Need is of learning from them, adding science to such modes if needed, robust evaluation of community actions with long term outcomes, cost-effectiveness, in various settings globally. World needs to know and use for healthy life and make communities aware of modern medicine, technology wherever essential for global health.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"131 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140369562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"An analysis of indices and ratios in anthropometric body measurements among team sports athletes","authors":"M. H. Rahman, J. P. Sharma","doi":"10.18203/issn.2454-2156.intjscirep20240714","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240714","url":null,"abstract":"Background: Physical indices and proportions based on anthropometry are crucial for determining the performance of players in team sports. The study aims to analyze the anthropometric status, body mass and shape indices, including waist-to-hip and height ratios, among athletes from different team sports. \u0000Methods: Study involved 135 male team-sport athletes specializing in basketball, handball, and volleyball, with 45 players in each sport. Researchers collected anthropometric data, including chronological age (CA), height (Ht), weight (Wt), body mass index (BMI), waist circumferences (WC) and hip circumferences (HC), waist-hip ratio (WHR), waist-height ratio (WHtR), and a body shape index (ABSI). To compare variables across groups, descriptive statistics and one-way ANOVA with LSD post-hoc tests used. Relationships within each group were examined using correlation analysis. \u0000Results: The ANOVA findings indicated f values (2, 132) for CA at 0.57, p=0.57, BMI at 0.59, p=0.56, WC at 2.10, p=0.13, WHR at 1.37, p=0.27, and ABSI at 1.41, p=0.25, respectively, revealing no significant disparities among the groups. Conversely, Ht at 13.36, p=0.00, Wt at 5.74, p=0.00, HC at 6.33, p=0.00, and WHtR at 3.60, p=0.03, respectively, highlighted significant variations between the groups. Furthermore, the correlation of BMI, WC, HC, WHR, WHtR, and ABSI in each group showed a strong positive correlation (p<0.01). \u0000Conclusions: Athletes in team sports were found to have similar body mass and shape indices, including ratios for WHR, while the WHtR ratio showed significant variations among the groups. Despite these differences, measures of athletes’ health metrics across these sports remain within safe, normal ranges.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"125 52","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140370095","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"A meta-analysis evaluating the role of calcium channel alpha-2 delta-1 subunit in carcinogenesis","authors":"C. Raybarman, Surajit Bhattacharjee","doi":"10.18203/issn.2454-2156.intjscirep20240715","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240715","url":null,"abstract":"There is hardly found any study accumulating all the experiments reported with the expression of alpha-2 delta-1 (α2δ-1) in cancer cells. This meta-analysis aimed to advance our knowledge about the role of calcium channel alpha2 delta-1 subunit in carcinogenesis in the present time. PubMed searches for peer-reviewed articles were conducted using the keywords “α2δ-1 protein in oncogenesis”, “α2δ-1 protein expression in cancer cells”, and “α2δ-1 protein as cancer cell marker”. The databases were developed in accordance with PRISMA guidelines. Seventeen studies out of 80 citations met the inclusion criteria pertaining to α2δ-1 expression in different cancer cells. The cancer patterns were hepatocellular carcinoma in 41%, non-small cell lung carcinoma in 12% and laryngeal squamous cell carcinoma in 12%. The remaining studies included small-cell lung cancer (6%), gastric cancer (6%), pancreatic cancer (6%), hypopharyngeal squamous cell carcinoma (6%), breast cancer (6%) and glioblastoma multiforme (6%). α2δ-1+ cells had a higher sphere-forming and tumorigenic efficiency in 76.5% of experiments. 58.8% experiments explored mechanistically in self-renewal efficiency and tumorigenesis of α2δ-1+ cancer cells. The cancer cells expressing α2δ-1 have the potential to serve as cell surface markers for tumour-initiating cells and cancer stem cells. These intriguing findings open up a promising avenue for future research, focusing on the targeting of α2δ-1 as a potential therapeutic strategy for cancer treatment.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"132 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140369719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Safa K. Salman, Dina I. Esmandar, Enana K. Sarem, Ram F. Attaf
{"title":"The prevalence of premenstrual syndrome symptoms and associated factors among female medical students at Al-Andalus University: a cross-sectional study","authors":"Safa K. Salman, Dina I. Esmandar, Enana K. Sarem, Ram F. Attaf","doi":"10.18203/issn.2454-2156.intjscirep20240713","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240713","url":null,"abstract":"Background: Premenstrual syndrome (PMS) is a menstrual disorder defined as a cluster of various physical, emotional, and behavioural symptoms that negatively affect women’s quality of life. This cross-sectional study aimed to assess the prevalence of common PMS symptoms and determine their association with body mass index (BMI), lifestyle, dietary habits, perceived stress, menstrual flow, family history and family income among medical students at Al-Andalus University in Syria.\u0000Methods: A sample of 205 female students were interviewed and filled out a questionnaire. All data were coded and entered into excel (Microsoft 2019). One-way analysis of variance (ANOVA) and Chi-square tests were used to evaluate the association between PMS symptoms and the studied factors. \u0000Results: The most frequently reported PMS symptoms were anxiety (80%), bloating (74.6%), and breast tenderness (62.9%). Our study found a significant association between weight gain pre-menstruation and BMI (p=0.02). A significant relationship was found between increased premenstrual consumption of sweets and both breast tenderness and bloating (p=0.01). Daily consumption of coffee was significantly correlated with weight gain and headaches (p<0.001). This study found a significant relationship between smoking cigarettes and abdominal and back pain (p=0.04, p=0.02, respectively). Smoking shisha was significantly associated with fatigue and bloating (p=0.01, p<0.001, respectively). A significant relationship was found between menstrual flow and both abdominal pain and headaches (p=0.01, p=0.02, respectively).\u0000Conclusions: A high prevalence of PMS symptoms was reported in Al-Andalus University female medical students. Additionally, this study found multiple significant associations between BMI, dietary habits, lifestyle, menstrual flow and the severity of symptoms.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"117 42","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140370056","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Trofinetide providing a promising avenue for the treatment of Rett syndrome","authors":"Raja Devendar, Anum Fatima Shigri, Maheera Khan","doi":"10.18203/issn.2454-2156.intjscirep20240718","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240718","url":null,"abstract":"Rett syndrome (RTT) is a developmental disability associated primarily with the abnormal functioning of the nervous system. Affecting 1 in 10,000-15,000 women, it results from an inactivating modification in the X-linked genetic code, methyl-CpG-binding protein 2 (MECP2), which codes for a protein that binds to DNA and regulates transcription. Girls with RTT are born with normal prenatal and perinatal periods and appear to be in good health. Their psychomotor growth is typical up to their first or second year of life, at which point brain functional regression starts. Clinical signs of the regression include the emergence of stereotyped hand movements, a loss of fine motor skills, gait apraxia, verbal and nonverbal communication deficits, and epileptic seizures.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"65 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140371608","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Amith Kiran, Swathi U. Shetty, Vishnu Keerthi B. S., Sharangoud Patil
{"title":"A rare case report of accessory thyroid in the superior mediastinum in the form of multinodular goiter","authors":"Amith Kiran, Swathi U. Shetty, Vishnu Keerthi B. S., Sharangoud Patil","doi":"10.18203/issn.2454-2156.intjscirep20240048","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240048","url":null,"abstract":"Retrosternal expansion of a goiter is one of the most common types of mass in the upper mediastinum. Although there is no clear definition of retrosternal, substernal, or mediastinal goiter in the literature, it usually refers to an extension of thyroid tissue from the cervical part that continues to the anterior mediastinum of the aortic arch. One of the interesting features of these cervical mediastinal lesions is that they may not be continuous. Such goiter types classically present with compression symptoms such as dyspnea, dysphonia, dysphagia, or sleep apnea, and less frequently, these masses can compress neurovascular structures, leading to superior vena cava (SVC) syndrome and Horner’s syndrome. In our case report, A 47-year-old female from North Karnataka presented to the hospital with history of dyspnea, dysphonia, and dysphagia persisting for the last 3 years. After careful examination and investigation, a diagnosis of thymoma was made initially. However, upon biopsy of the excised tumor, unexpected finding was revealed, it was identified as a multinodular goiter (MNG) rather than a thymoma.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"27 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139602207","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
N. Mabika, Rachel Gwazani, Musara Chipumuro, Tsitsi Zengeya, Lawrence Sawunyama
{"title":"A preliminary assessment of the length-weight relationship and condition factor of Hydrocynus vittatus and Oreochromis niloticus in Lake Kariba, Zimbabwe","authors":"N. Mabika, Rachel Gwazani, Musara Chipumuro, Tsitsi Zengeya, Lawrence Sawunyama","doi":"10.18203/issn.2454-2156.intjscirep20240046","DOIUrl":"https://doi.org/10.18203/issn.2454-2156.intjscirep20240046","url":null,"abstract":"Background: Hydrocynus vittatus and Oreochromis niloticus are two economically important fish species in Zimbabwe. The length-weight relation can be employed to assess the health and well-being of fish so as to assist fish farmers. The objective of the study was to investigate the length-weight relationship and condition factor of these fish species.\u0000Methods: Thirty-four fish of each species (Hydrocynus vittatus and Oreochromis niloticus) were collected by seine netting in the Sanyati Basin, Lake Kariba in August 2019. The total length (TL) and standard length (SL) were measured in centimetre (cm), and the body weight (BW) was measured in grams (g). The body weight (BW) was measured using a digital top-loading electronic weighing balance. \u0000Results: The value of the exponent b in the LWR for H. vittatus was 2.984, indicating negative allometry (b<3). The exponent value of O. niloticus in the LWR was 3.04 (b>3) implying positive allometry. The r2 values for H. vittatus and O. niloticus were 92.3% and 97.3% respectively, indicating that there was a high degree of positive correlation. The condition factor for both fish species were greater than 1.60 implying good physiological conditions for both fish species.\u0000Conclusions: This study provides preliminary information on length-weight parameters of O. niloticus whose data was previously not available in the take.","PeriodicalId":14297,"journal":{"name":"International Journal of Scientific Reports","volume":"19 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139601556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}