Good Therapeutic Outcome Research Articles

The Contribution of Shame to Eating Disorder Treatment Outcomes in a Community Mental Health Clinic.

Shame is a powerful self-conscious emotion that is often experienced by individuals with eating disorders (EDs). While the association between EDs and shame is well-established, there is limited research investigating the contribution of pre-treatment shame to clinical outcomes. Participants (N = 273) received outpatient cognitive-behavioral therapy for eating disorders (CBT-ED). We investigated pre-treatment shame as a predictor of dropout and as a moderator of change in ED psychopathology and clinical impairment from pre-treatment to post-treatment. We also explored the potentially moderating roles of body mass index, ED diagnostic category, and co-occurring anxiety and depression diagnoses. Shame improved substantially (d = 1.28) despite not being explicitly targeted in treatment. Pre-treatment shame did not predict treatment dropout. Individuals high in shame started and ended treatment with higher ED symptoms and impairment than those with low shame. The contribution of pre-treatment shame on the degree of change in symptoms/impairment depended critically on whether analyses controlled for pre-treatment symptoms/impairment. When those were controlled, high pre-treatment shame was associated with substantially less improvement in ED symptoms and impairment. There was some evidence that ED diagnosis and co-occurring depressive diagnoses may moderate the relationship between shame and treatment outcome. Changes in shame were positively associated with changes in ED symptoms and clinical impairment. A high level of shame at pre-treatment is not a contraindication for CBT-ED as good therapeutic outcomes can be achieved. However, outcomes may be enhanced among individuals high in shame by offering adjunctive interventions that explicitly target shame.

PdCu nanoparticles with multienzymatic activity to treat tumors through the synergistic photothermal and chemodynamic therapy

Combined chemodynamic/photothermal therapy has great potential in tumor treatment. However, the presence of excessive glutathione (GSH) in the tumor microenvironment (TME) can attenuate its therapeutic effect, and other components in the TME have not been fully utilized as well. In this article, we designed a noble metal nanozyme called PdCu@BSA, which can be used for the combined chemodynamic therapy (CDT) and photothermal therapy (PTT) of tumor. In detail, PdCu@BSA has three different types of enzyme-like activities. Its catalase (CAT)-like activity can degrade extra H2O2 in the TME to create O2 and relieve the hypoxic situation. The glutathione oxidase (GSHox)-like activity can consume high level of GSH in the TME to reduce the consumption of reactive oxygen species (ROS). Peroxidase (POD)-like activity catalyzes H2O2 to form strong oxidized ·OH. The above enzyme-like activities enhance the effectiveness of CDT. Besides, PdCu@BSA has good photothermal effect and can be used for PTT when exposed to 1064 nm laser. Therefore, based on multiple enzyme-like activities and photothermal effects, PdCu@BSA can be employed for synergistic tumor therapy, resulting in good therapeutic outcome.

Comparative Efficacy and Safety of Potassium-Competitive Acid Blockers vs. Proton Pump Inhibitors for Peptic Ulcer with or without Helicobacter pylori Infection: A Systematic Review and Network Meta-Analysis.

Novel potassium-competitive acid blockers (P-CABs) have emerged as effective acid-suppressive drugs in recent years, replacing proton pump inhibitors (PPIs). We aim to compare the efficacy and safety of P-CABs versus PPIs in the treatment of peptic ulcers with or without Helicobacter pylori (H. pylori) infection. We searched in PubMed, Embase, WOS, Cochrane Library, ClinicalTrials.gov, CNKI, and Wanfang databases (all years up to January 2024). Efficacy and safety outcomes were evaluated using odds ratio (OR) and 95% confidence intervals (CI). The Surface Under the Cumulative Ranking (SUCRA) probabilities were used to rank each intervention. Among 14,056 studies screened, 56 studies involving 9792 participants were analyzed. Vonoprazan demonstrated the best efficacy in ulcer healing rate and H. pylori eradication rate (SUCRA = 86.4% and 90.7%, respectively). Keverprazan ranked second in ulcer healing rates (SUCRA = 76.0%) and was more effective in pain remission rates (SUCRA = 91.7%). The risk of adverse events was low for keverprazan (SUCRA = 11.8%) and tegoprazan (SUCRA = 12.9%), and moderate risk for vonoprazan (SUCRA = 44.3%) was demonstrated. Compared to lansoprazole, vonoprazan exhibited a higher risk of drug-related adverse events (OR: 2.15; 95% CI: 1.60-2.89) and serious adverse events (OR: 2.22; 95% CI: 1.11-4.42). Subgroup analysis on patients with H. pylori-positive peptic ulcers showed that vonoprazan was at the top of the SUCRA rankings, followed by keverprazan. Vonoprazan showed superior performance in peptic ulcers, especially for patients with H. pylori-positive peptic ulcers. However, the risk of adverse events associated with vonoprazan should be noted. Keverprazan has also shown good therapeutic outcomes and has performed better in terms of safety.

Analysis of the Effectiveness of Modified Non-Traumatic Filling Technology in the Treatment of Pediatric Dental Caries

Objective: To analyze the effectiveness of modified non-traumatic filling technology in the treatment of pediatric dental caries. Methods: Ninety-seven children with dental caries who were treated in our hospital (Panyu Maternal and Child Care Service Center of Guangzhou) from January 2022 to December 2023 were selected and randomly divided into two groups, with 48 cases in the experimental group and 49 in the control group. The experimental group was treated with modified non-traumatic filling techniques, while the control group was treated with conventional filling techniques. Observation indicators such as the total effectiveness of the treatment, incidence of adverse events, treatment compliance, and pain scores were analyzed after the intervention. Result: After intervention, the total effectiveness of the treatment in the experimental group was higher than that in the control group (P < 0.05). The incidence of adverse events in the experimental group was lower than that of the control group (P < 0.05). The treatment compliance of patients in the experimental group was higher than that in the control group (P < 0.05). The pain score of the experimental group was lower than that of the control group (P < 0.05). Conclusion: The application of modified non-traumatic filling technology in the treatment of pediatric dental caries showed good therapeutic outcomes. After the intervention, the child’s symptoms were significantly alleviated, the incidence of adverse events such as filling material falling off was reduced, their compliance was improved, and the pain was relieved. This procedure is worth to be promoted for clinical application.

Examining co-occurring social anxiety in cognitive behavioral therapy for eating disorders: Does it change and does it moderate eating disorder outcomes?

Eating disorders (EDs) often co-occur with social anxiety disorder (SAD). However, little research has examined the influence of SAD symptoms on ED treatment outcomes in the context of individual outpatient cognitive-behavior therapy for eating disorders (CBT-ED). It is plausible that SAD symptom severity could improve as a result of ED treatment, given the high overlap between EDs and SAD. We sought to test whether baseline SAD symptoms moderate early response to CBT-ED or post-treatment outcomes in CBT-ED, and the degree to which SAD symptoms improve during therapy despite SAD not being an explicit treatment target. ED clients (N = 226) aged ≥16 years were treated with CBT-ED. Outcomes were ED symptoms, clinical impairment, and SAD symptoms measured at baseline, session 5 and post-treatment. Baseline SAD was a weak moderator of early and post-treatment ED symptoms and impairment. SAD symptoms improved moderately over treatment among clients who started with elevated levels of SAD symptomology. Clients with EDs can experience good therapeutic outcomes regardless of their social anxiety severity at pre-treatment. SAD symptoms reduced over CBT-ED, but protocol enhancements such as exposure-based strategies that directly target co-occurring social-evaluative concerns may help achieve larger reductions in SAD symptoms. Eating disorders often co-occur with anxiety disorders such as social anxiety. We found people who had both social anxiety and an eating disorder benefited as much from eating disorder treatment as people who did not have social anxiety. People who were socially anxious became less anxious as a by-product of receiving eating disorder treatment. It may be possible to reduce social anxiety further by enhancing eating disorder treatment protocols.

Inter‐modality comparison of working alliance and client satisfaction in online counselling during the COVID‐19 pandemic: An exploratory study

AbstractPurposeMental health suffered during the COVID‐19 pandemic lockdown in Scotland. While there was limited availability for face‐to‐face counselling, online counselling was available via telephone, videoconference, email, one‐to‐one chat and group basis. Existing research has failed to examine inter‐modality comparisons of client experiences of online counselling during the COVID‐19 pandemic.MethodsScottish participants (n = 94) who received online counselling during the COVID‐19 pandemic completed an online survey using quantitative methodology, which explored their experiences of one of five online counselling modalities measured by two reliable scales: the Working Alliance Inventory‐Short Revised and factor one of the Satisfaction with Therapy and Therapist Scale. Additional exploratory analysis investigated whether age, gender, choice of online counselling modality and pre‐COVID‐19 pandemic familiarity with online counselling affected these outcomes.ResultsWorking alliance and satisfaction outcomes were positively correlated. No significant differences between working alliance and satisfaction scores were found between the five modalities. Female participants, and those who received their preferred choice of counselling modality, had significantly greater working alliance than those who did not.ConclusionThese findings provide insights to clients and practitioners about the variety of synchronous and asynchronous online platforms that can facilitate good therapeutic outcomes. The importance of participant choice of counselling modality was reiterated. The broader implications of the findings and the future direction of a hybrid online and face‐to‐face counselling model were discussed.

Cholangitis with bacteremia due to Pseudomonas nitroreducens in a patient with pancreatic neuroendocrine tumors: a case report

BackgroundPseudomonas nitroreducens is a non-fermenting, gram-negative, rod-shaped bacterium commonly inhabiting soil, particularly soil contaminated with oil brine. To our knowledge, no cases of human infection with P. nitroreducens have been previously reported. Here, we present the first documented case of cholangitis caused by P. nitroreducens in a patient with bacteremia.Case presentationA 46-year-old Japanese man with an advanced pancreatic neuroendocrine tumor was hospitalized with fever and chills. Four days before admission, the patient developed right upper abdominal pain. Two days later, he also experienced fever and chills. Endoscopic retrograde cholangiopancreatography was performed on the day of admission, and the patient was diagnosed as having cholangitis associated with stent dysfunction. Gram-negative rods were isolated from blood cultures, but attempts to identify the bacteria using VITEK2 and matrix-assisted laser desorption/ionization time-of-flight mass spectrometry (MALDI-TOF MS) with VITEK MS ver. 4.7.1 (bioMérieux Japan Co. Ltd., Tokyo, Japan) were unsuccessful. Finally, the organism was identified as P. nitroreducens using MALDI-TOF MS with a MALDI Biotyper (Bruker Daltonics Co., Ltd., Billerica, MA, USA) and 16 S ribosomal RNA sequencing. Despite thorough interviews with the patient, he denied any exposure to contaminated soil. The patient was treated with intravenous cefepime and oral ciprofloxacin for 16 days based on susceptibility results, achieving a good therapeutic outcome. At the outpatient follow-up on day 28, the patient was in good general condition.ConclusionsThis is the first reported human case of cholangitis with bloodstream infection caused by P. nitroreducens. This report provides clinicians with novel insights into the clinical manifestations and diagnostic methods necessary for the accurate diagnosis of P. nitroreducens, along with guidance on treatment.

The effect of velar collapse patterns on unilateral upper airway stimulation therapy.

Hypoglossal nerve stimulation is a promising alternative therapy for patients with obstructive sleep apnea with continuous positive airway pressure intolerance or failure. Previous studies concluded that a velar complete concentric collapse might prohibit a good therapeutic outcome. However, certain patients have an upper velar anteroposterior collapse and a lower velar complete concentric collapse. The effect of this velar collapse pattern is unknown, preventing evidence-based decision-making for these patients. This study aimed to compare the results of upper airway stimulation therapy in these patients to patients with a pure anteroposterior velar collapse. A retrospective single-center cohort study was performed. Patients were included who were implanted with an upper airway stimulation device and had a 1-year follow-up. Of 66 patients, 10 had an upper velar anteroposterior collapse and lower velar complete concentric collapse. Fifty-six patients had a complete or partial velar anteroposterior collapse. At follow-up, all respiratory outcomes were similarly changed between the two groups. The mean apnea and hypopnea index reduced equally (26.9 events/hour vs. 23.9 events/hour, 95% CI (-5.0, 11.0), p = 0.46). A similar decrease in the oxygen desaturation index of ≥ 4% was observed (12.0/hour versus 11.5/hour, 95% CI (-8.7, 9.7) p = 0.92) CONCLUSION: Patients with an upper velar anteroposterior collapse and a lower velar complete concentric collapse are suitable candidates for upper airway stimulation therapy. In these patients, the lower velum may represent a transition zone between the anteroposterior collapse of the upper velum and the lateral collapse of the oropharynx, instead of being a real concentric collapse.

The use of mentalization-based techniques in online psychodynamic child psychotherapy

Objective: Psychodynamic child psychotherapy is an evidence-based approach for a range of child mental health difficulties and needs to constantly adapt to meet the needs of children. This study is the first to investigate whether the use of mentalization-based interventions (i.e., a focus on promoting attention control, emotion regulation, and explicit mentalization) predicted a good therapeutic outcome in online psychodynamic child therapy sessions conducted during the COVID-19 pandemic. Methods: The sample included 51 Turkish children (M age = 7.43, 49% girls) with mixed emotional and behavioral problems. Independent raters coded 203 sessions from different phases in each child’s treatment using the Mentalization-Based Treatment for Children Adherence Scale (MBT-CAS). Results: Multilevel modeling analyses showed children with higher emotional lability benefited more from attention control interventions compared to those with lower emotional lability. Discussion: Interventions that focus on developing the basic building blocks of mentalizing may be effective components of therapeutic action for online delivery of psychodynamic child psychotherapy, especially for children with greater emotional lability.

A protocol for randomized controlled trial on multidisciplinary interventions for mobility limitation in the older adults (M-MobiLE)

BackgroundMobility limitation—the loss of exercise capacity or independent living ability—is a common geriatric syndrome in older adults. As a potentially reversible precursor to disability, mobility limitation is influenced by various factors. Moreover, its complex physiological mechanism hinders good therapeutic outcomes with a single-factor intervention. Most hospitals have not incorporated the diagnosis and evaluation of mobility limitation into medical routines nor developed a multidisciplinary team (MDT) treatment plan. We aim to conduct a clinical trial titled “A Multidisciplinary-team approach for management of Mobility Limitation in Elderly (M-MobiLE)” to explore the effect of the MDT decision-making intervention for mobility limitation.MethodsThe M-MobiLE study will be a multicenter, randomized, and controlled trial. We will recruit a minimum of 66 older inpatients with mobility limitation from at least five hospitals. Older patients with mobility limitation admitted to the geriatrics department will be included. Short-Physical Performance Battery (SPPB), Activities of Daily Living (ADL), Function Impairment Screening Tool (FIST), Geriatric Depression Scale (GDS-15), Short Form − 12 (SF-12), Fried frailty phenotype, social frailty, Morse Fall Risk Scale, SARC-CalF, Mini-Mental State Examination (MMSE), Mini-Nutritional Assessment Short-Form (MNA-SF), and intrinsic capacity will be assessed. The intervention group will receive an exercise-centered individualized MDT treatment, including exercise, educational, nutritional, medical, and comorbidity interventions; the control group will receive standard medical treatment. The primary outcome is the change in the SPPB score, and the secondary outcomes include increased SF-12, ADL, FIST, MMSE, MNA-SF, and intrinsic capacity scores and decreased GDS-15 and SARC-CalF scores.ConclusionOur results will help develop a multidisciplinary decision-making clinical pathway for inpatients with mobility limitation, which can be used to identify patients with mobility limitation more effectively, improve mobility, and reduce the risk of falls, frailty, and death in older inpatients. The implementation of this MDT strategy may standardize the treatment of mobility limitation, reduce adverse prognosis, and improve quality of life.Trial registrationChiCTR, ChiCTR2200056756, Registered 19 February 2022.

Association between Ki67 expression and therapeutic outcome in colon cancer.

Ki67 is a commonly used proliferation marker in pathological diagnosis of tumors; however, its prognostic value in colon cancer is controversial. A total of 312 consecutive patients with stage I-III colon cancer who underwent radical surgery with or without adjuvant chemotherapy were included in the present study. Ki67 expression was assessed using immunohistochemistry and was classified according to 25% intervals. The association between Ki67 expression and clinicopathological features was analyzed. Long-term postoperative survival, including disease-free survival (DFS) and overall survival, was calculated, and its association with Ki67 was analyzed. High Ki67 expression (>50%) was associated with improved DFS in patients treated with adjuvant chemotherapy postoperatively, but not in patients who received surgery alone (P=0.138). Ki67 expression was significantly associated with histological differentiation of the tumor (P=0.01), while it was not associated with other clinicopathological factors. Multivariate analysis demonstrated that pathological T and N stage were independent prognostic factors. In conclusion, high Ki67 expression was associated with a good therapeutic outcome in patients receiving adjuvant chemotherapy in colon cancer.

Vaginismus: An Approach from biology to psychological aspect

Background: The prevalence of vaginismus ranges from 1-6%, and the ratio becomes 5%-17% in clinical setting, although it varies widely worldwide. The main cause of vaginismus is not known, but is often considered multifactorial. This paper aims to describe the factors that contribute to vaginismus. Case: A 26 years old, married woman, health practitioner presented with the complaint of not being able to have sexual intercourse. About 1 month ago, since marriage, the patient was unable to have sexual intercourse with her husband. The patient feels guilty and anxious because of the inability to please her husband. The patient's father was very dominant and was a "religious" type of parent who always gave advice with religious aspects regarding all matters. The patient's mother was a nervous person and always said that what the father said was a law that had to be obeyed. Likewise, talk about sex is a taboo topic of discussion and is considered embarrassing. Discussion: Many factors need to be explored related vaginismus. In the case, the biological aspect can be ruled out because the patient has consulted with the ob-gyn doctor. from the psychological aspect; perceptions, attitudes and attitudes towards sex and the values ​​held by parents contribute to vaginismus in this patient. Conclusion: a therapeutic approach which focuses on all aspects promises quite good therapeutic outcomes.

Development of a sorafenib-loaded solid self-nanoemulsifying drug delivery system: Formulation optimization and characterization of enhanced properties.

Sorafenib, marketed under the brand name Nexavar®, is a multiple tyrosine kinase inhibitor drug that has been actively used in the clinical setting for the treatment of several cancers. However, the low solubility and bioavailability of sorafenib constitute a significant barrier to achieving a good therapeutic outcome. We developed a sorafenib-loaded self-nanoemulsifying drug delivery system (SNEDDS) formulation composed of capmul MCM, tween 80, and tetraglycol, and demonstrated that the SNEDDS formulation could improve drug solubility with excellent self-emulsification ability. Moreover, the sorafenib-loaded SNEDDS exhibited anticancer activity against Hep3B and KB cells, which are the most commonly used hepatocellular carcinoma and oral cancer cell lines, respectively. Subsequently, to improve the storage stability and to increase the possibility of commercialization, a solid SNEDDS for sorafenib was further developed through the spray drying method using Aerosil® 200 and PVP K 30. X-ray diffraction and differential scanning calorimeter data showed that the crystallinity of the drug was markedly reduced, and the dissolution rate of the drug was further improved in formulation in simulated gastric and intestinal fluid conditions. In vivo study, the bioavailability of the orally administered formulation increases dramatically compared to the free drug. Our results highlight the use of the solid-SNEDDS formulation to enhance sorafenib's bioavailability and outlines potential translational directions for oral drug development.

Diagnosis and management of lip filler complications – a case study with literature review

Introduction 
 Aesthetic medicine treatments are becoming more popular in the 21st century due to the changing beauty standards and the treatments’ ability to help slow down the aging process. One of the most asked for treatments is a lip filler using hyaluronic acid. The popularity and frequency of the said treatment are directly connected to the rising number of complications in this area. The right diagnosis of lip filler complications allows to implement correct management and therefore therapeutic success for the patient.
 The aim 
 The aim of the work was to analyze and discuss patient’s complications and implemented medical management after lip filler treatment using hyaluronic acid.
 Case report
 An individual case study that included clinical examination, medical and photographic documentation analysis.
 Results
 The patient was diagnosed with one of the most common complications after lip filler which is swelling. The swelling was a result of an allergic reaction and was classified as severe. Medical management using corticosteroid and antihistamines was implemented with a good therapeutic outcome.
 Conclusions
 Implemented management using corticosteroids and antihistamines for severe swelling after lip filler is effective. Due to the rising number of treatments using soft tissue fillers, it is expected that more patients will present with complications after aesthetic medicine procedures.

Symphony in the crowd: Key genetic alterations in prostate cancer.

Androgen receptor (AR) signaling have been frequently targeted for treating prostate cancer (PCa). Even though primarily patients receive a good therapeutic outcome by targeting AR signaling axis, eventually it emerges resistance by altering the genetic makeup of prostate cells. However, to develop an effective therapeutic regime, it is essential to recognize key genetic alterations in PCa. The most common genetic alterations that give rise to distinct androgen different differentiation states are gene fusion of TMPRSS2 with ETS family genes, deletion, or mutation of tumor suppressor PTEN and TP53 gene, amplification or splicing of AR, altered DNA repair genes. In this review, we describe key genes and genetic changes that have been recognized to contribute to altered prostate environment.

Seaweed-Derived Sulfated Polysaccharides; The New Age Chemopreventives: A Comprehensive Review.

Seaweed-derived bioactive compounds are regularly employed to treat human diseases. Sulfated polysaccharides are potent chemotherapeutic or chemopreventive medications since it has been discovered. They have exhibited anti-cancer properties by enhancing immunity and driving apoptosis. Through dynamic modulation of critical intracellular signalling pathways, such as control of ROS generation and preservation of essential cell survival and death processes, sulfated polysaccharides' antioxidant and immunomodulatory potentials contribute to their disease-preventive effectiveness. Sulfated polysaccharides provide low cytotoxicity and good efficacy therapeutic outcomes via dynamic modulation of apoptosis in cancer. Understanding how sulfated polysaccharides affect human cancer cells and their molecular involvement in cell death pathways will showcase a new way of chemoprevention. In this review, the significance of apoptosis and autophagy-modulating sulfated polysaccharides has been emphasized, as well as the future direction of enhanced nano-formulation for greater clinical efficacy. Moreover, this review focuses on the recent findings about the possible mechanisms of chemotherapeutic use of sulfated polysaccharides, their potential as anti-cancer drugs, and proposed mechanisms of action to drive apoptosis in diverse malignancies. Because of their unique physicochemical and biological properties, sulfated polysaccharides are ideal for their bioactive ingredients, which can improve function and application in disease. However, there is a gap in the literature regarding the physicochemical properties and functionalities of sulfated polysaccharides and the use of sulfated polysaccharide-based delivery systems in functional cancer. Furthermore, the preclinical and clinical trials will reveal the drug's efficacy in cancer.

Postoperative radiotherapy timing, molecular subgroups and treatment outcomes of Thai pediatric patients with medulloblastoma.

Medulloblastoma (MB) is the most common childhood malignant brain tumor worldwide. Recently, molecular classification was established and started to play a role in the management of MB; however, studies involving molecular defined MB in Southeast Asia have been limited. We aimed to describe, and correlate clinical characteristics and molecular subgroups with therapeutic outcomes of Thai pediatric patients with MB. Pediatric MB patients treated at King Chulalongkorn Memorial Hospital in Thailand from 2006 to 2018 were recruited. Patients were classified by clinical characteristics into standard- and high-risk groups, which determined treatment regimen. Retrospectively, available tumor tissues were classified into 3 molecular subgroups using immunohistochemistry: 1) WNT, 2) SHH, and 3) non-WNT/non-SHH. The primary outcome was 5-year overall survival (OS). Risk factors associated with OS were analyzed using cox regression analysis. Fifty-three Thai pediatric patients with MB were enrolled. The median follow-up time was 60 months. The 5-year OS for all patients, and patients with standard-risk and high-risk were 74.2%, 76.3% and 71.4%, respectively. Tumor tissues of 24 patients were available, of which 23 could be molecularly classified. Two, one and 20 were in the WNT, SHH and non-WNT/non-SHH subtypes with 5-year OS of 100%, 100% and 78.9%, respectively. Using multivariate analysis, the interval of more than 8 weeks between surgery and radiotherapy was significantly correlated with a decrease in the 5-year OS. Interval between surgery and radiotherapy within 8 weeks was associated with good therapeutic outcomes among Thai pediatric patients with MB. Simplified molecular subtyping combined with clinical characteristics is practical in risk classification of patients with MB in institutes with limited resources.

Gene therapy for lysosomal storage diseases: Current clinical trial prospects.

Lysosomal storage diseases (LSDs) are a group of metabolic inborn errors caused by defective enzymes in the lysosome, resulting in the accumulation of undegraded substrates. LSDs are progressive diseases that exhibit variable rates of progression depending on the disease and the patient. The availability of effective treatment options, including substrate reduction therapy, pharmacological chaperone therapy, enzyme replacement therapy, and bone marrow transplantation, has increased survival time and improved the quality of life in many patients with LSDs. However, these therapies are not sufficiently effective, especially against central nerve system abnormalities and corresponding neurological and psychiatric symptoms because of the blood-brain barrier that prevents the entry of drugs into the brain or limiting features of specific treatments. Gene therapy is a promising tool for the treatment of neurological pathologies associated with LSDs. Here, we review the current state of gene therapy for several LSDs for which clinical trials have been conducted or are planned. Several clinical trials using gene therapy for LSDs are underway as phase 1/2 studies; no adverse events have not been reported in most of these studies. The administration of viral vectors has achieved good therapeutic outcomes in animal models of LSDs, and subsequent human clinical trials are expected to promote the practical application of gene therapy for LSDs.

Clinical prolife and outcome in COVID-19 patients on treatment regimen of remdesivir, favipiravir, and non-antiviral drugs

Background: Although various drugs have been used in the treatment of COVID-19 infections, specific drug with good therapeutic outcome is still unclear. They are using remdesivir, favipiravir, ivermectin, azithromycin, and so on. Aim and Objectives: This study aimed to evaluate the clinical profile of patients treated with different regimens and their outcome in improvement or morbidity of patients. Materials and Methods: We conducted a cross-sectional questionnaire-based study after approval by the institutional ethics committee. Data were collected from case files of COVID-19-positive patients treated in COVID-19 ward in Bharati Hospital, Sangli. We collected as per our protocol pro forma as – routine COVID treatment with remdesivir, favipiravir, and non-antiviral drugs. We analyzed various clinical profiles (presenting complaints), duration of stay in COVID wards, their outcome as improved and discharged, or deaths occurred. Results: It was observed that majority 54% of patients were from 40 to 50 years’ range. There was male preponderance 64%. Clinical symptoms were fever (92%), cough (85%), sore throat (77%), headache (53%), and breathlessness (10%). Majority of them were diabetes mellitus (51%), obesity (22%), and hypertension. Ventilatory support needed in 18%, rest recovered on pharmacotherapy. Conclusion: One of the highly infectious diseases causing COVID-19 is contagious and transmitted through airborne droplets generated by sneezing, smooching, and coughing. So as per the WHO and CDC guidelines avoid close contact with these infected persons in a market or public place. Vaccines available against COVID-19 Covishield and Covaxin to prevent the severity and to have immunity against COVID should be taken by all.

Neurotoxins Acting at Synaptic Sites: A Brief Review on Mechanisms and Clinical Applications.

Neurotoxins generally inhibit or promote the release of neurotransmitters or bind to receptors that are located in the pre- or post-synaptic membranes, thereby affecting physiological functions of synapses and affecting biological processes. With more and more research on the toxins of various origins, many neurotoxins are now widely used in clinical treatment and have demonstrated good therapeutic outcomes. This review summarizes the structural properties and potential pharmacological effects of neurotoxins acting on different components of the synapse, as well as their important clinical applications, thus could be a useful reference for researchers and clinicians in the study of neurotoxins.