anthonyyazdaniml/gliner-biomed-curated-corpus huggingface dataset:
GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid). It is an extremely rare progressive muscle disease, often occurring in the late teens to 30s, usually affecting the distal lower leg, especially the extensor muscles such as the tibialis anterior muscle, first, and weakness in both upper and lower limbs. However, the weakness of the quadriceps femoris is usually gradual. Although there are large individual differences, health management and deterioration of QOL will eventually become a problem, and in severe cases, constant assistance is required in daily life.

A double-blind comparative study conducted in Japan provided results suggesting efficacy, but the efficacy could not be confirmed in a large-scale international clinical trial, so this study is decided to be conducted. Oral administration of aceneuramic acid tablets 500 mg (SA-ER tablets) or placebo tablets of the same appearance, 4 tablets at a time, 3 times a day for 48 weeks will be examined for differences in efficacy. The target was a total of 10 cases, 7 cases in the active drug group, and 3 in the placebo group. The amount of change in upper extremity composite score (the sum of the average of the right and left HHD scores for grip, shoulder abductors, elbow flexors and elbow extensors) was used, and the secondary endpoints were the doctor's comprehensive evaluation and the GNE myopathy functional activity scale.
Primary liver cancer is the second most common cause of cancer death in China, and hepatocellular carcinoma (HCC) represents more than 80% of liver cancer cases. The majority of HCC cases present at an advanced stage, and are thus not amenable to surgery. Immunotherapies, including PD-1 and PD-L1 inhibitors, alone or combined with other regimens, have shown encouraging clinical activity in unresectable HCC. Camrelizumab, a humanised monoclonal antibody against PD-1, has been shown to block the binding of PD-1 to PD-L1 and consequently inhibit the immune escape of tumour cells，which has been approved for the second-line treatment of advanced HCC. This multi-center, open-label, observational study aims to evaluate the safety and efficacy of camrelizumab in Chinese unresectable HCC in the real world. This study also aims to evaluate the treatment of camrelizumab in subgroups, to provide information of treatment regimens and efficacy in clinical practice. The anticipated sample size is 1000, and the camrelizumab-based treatment will be determined by the investigator.
Therapy-related myeloid neoplasm (t-MN), characterized by its association with prior exposure to cytotoxic therapy, remains poorly understood and is a major impediment to long-term survival even in the era of novel targeted therapies due to its aggressive nature and treatment resistance. Previously, cytotoxic therapy-induced genomic changes in hematopoietic stem cells were considered sine qua non in pathogenesis; however, recent research demonstrates a complex interaction between acquired and hereditary genetic predispositions, along with a profoundly senescent bone marrow (BM) microenvironment. We review emerging data on t-MN risk factors and explore the intricate interplay among clonal hematopoiesis, genetic predisposition, and the abnormal BM microenvironment. Significance: t-MN represents a poorly understood blood cancer with extremely poor survival and no effective therapies. We provide a comprehensive review of recent preclinical research highlighting complex interaction among emerging therapies, hereditary and acquired genetic factors, and BM microenvironment. Understanding the risk factors associated with t-MN is crucial for clinicians, molecular pathologists, and cancer biologists to anticipate and potentially reduce its incidence in the future. Moreover, better understanding of the molecular pathogenesis of t-MN may enable preemptive screening and even intervention in high-risk patients.
FIELD: medicine, cardiology. SUBSTANCE: the method deals with prescription of capotene during the first day of AMI at the background of standard therapy. In arterial hypertension-free patients at HF-III by Killip's capotene dosage should correspond to 0.12-0.20 mg/kg body weight daily. In case of arterial hypertension 130- 160/94 mm mercury column and HF II-III class by Killip's the dose of prescribed capotene is 0.21-0.29 mg/kg body weight daily. At arterial hypertension being above 160/94 mm mercury column and HF II-III class by Killip's capotene is prescribed at the dosage of 0.71- 1.190 mg/kg body weight daily. The method enables to provide an adequate myocardial protection during the first day of acute myocardial infarction. EFFECT: decreased number of complications. 1 dwg, 2 tbl
BACKGROUND: The association of genital Mollicutes infection transition with adverse pregnancy outcomes was insignificant among general pregnant women, but there remains a paucity of evidence linking this relationship in gestational diabetes mellitus (GDM) women. The aim was to investigate the association between genital Mollicutes infection and transition and adverse pregnancy outcomes in GDM women, and to explore whether this association still exist when Mollicutes load varied.
METHODS: We involved pregnant women who attended antenatal care in Chongqing, China. After inclusion and exclusion criteria, we conducted a single-center cohort study of 432 GDM women with pregnancy outcomes from January 1, 2018 to December 31, 2021. The main outcome was adverse pregnancy outcomes, including premature rupture of membrane (PROM), fetal distress, macrosomia and others. The exposure was Mollicutes infection, including Ureaplasma urealyticum (Uu) and Mycoplasma hominis (Mh) collected in both the second and the third trimesters, and testing with polymerase chain reaction method. The logistic regression models were used to estimate the relationship between Mollicutes infection and adverse pregnancy outcomes.
RESULTS: Among 432 GDM women, 241 (55.79%) were infected with genital Mollicutes in either the second or third trimester of pregnancy. At the end of the pregnancy follow-up, 158 (36.57%) participants had adverse pregnancy outcomes, in which PROM, fetal distress and macrosomia were the most commonly observed adverse outcomes. Compared with the uninfected group, the Mollicutes (+/-) group showed no statistical significant increase in PROM (OR = 1.05, 95% CI:0.51 ∼ 2.08) and fetal distress (OR = 1.21, 95% CI: 0.31 ∼ 3.91). Among the 77 participants who were both Uu positive in the second and third trimesters, 38 participants presented a declined Uu load and 39 presented an increased Uu load. The Uu increased group had a 2.95 odds ratio (95% CI: 1.10~8.44) for adverse pregnancy outcomes.
CONCLUSION: Mollicutes infection and transition during trimesters were not statistically associated with adverse pregnancy outcomes in GDM women. However, among those consistent infections, women with increasing Uu loads showed increased risks of adverse pregnancy outcomes. For GDM women with certain Mollicutes infection and colonization status, quantitative screening for vaginal infection at different weeks of pregnancy was recommended to provide personalized fertility treatment.
Over 30 million people in the US have diabetes. It's well known that acute inflammation can temporarily increase blood sugar levels (hyperglycemia) and insulin resistance among patients with diabetes, particularly those with type 1 diabetes (T1D). Vaccines cause an inflammatory reaction, because the vaccines prime the body's immune system against a potential virus. Reports have described temporary hyperglycemia and/or insulin resistance after COVID vaccine administration. However, to date this has not been studied in a rigorous fashion. The objective of the study is to determine if the COVID-19 booster causes increased sugar levels and insulin dose adjustments in patients with T1D. This is a study in adult patients with Type 1 Diabetes who have not yet received the COVID-19 booster vaccine. The study consists of two short outpatient (clinic) visits. Visit 1 occurs 3-5 days before receiving the COVID-19 booster vaccine (pre-vaccine visit). During this visit, the participant will fill out a questionnaire and have a blinded Dexcom G6 Pro CGM applied to the participant. An optional second "backup" Dexcom G6 Pro CGM will be provided, in case the first continuous glucose monitor (CGM) falls off accidentally. Visit 2 occurs 10 days after Visit 1 (about 5 days after receiving the first or second booster vaccine shot), at which time the CGM would be removed. Participants will continue participants' usual routine diabetes care while participating in the study, including using the participants' own glucometer or CGM. Participants will also be asked to keep track of participants' insulin doses administered during the 10-day study.
Participants will undergo a 3-day experimental paradigm. Before the experiment, participants will undergo a resting-state fMRI scan to determine the specific anatomical location of the TMS target for that particular individual (day 1). The resting-state scan will be about 10 minutes. Functional MRI scans will occur on days 1, 2, 3, and TMS on day 2. A blood draw will occur on experimental day 1 or, if needed due to scheduling, on any of the other experimental visits.
No community based Ag RDT testing but there has routine ATK or RT-PCR test by Thai health authorities if local health volunteer finds symptomatic or suspected COVID-19 or close contact with positive cases in their villages. The participant also involves in awareness-raising campaign with mask distribution.
The disclosure provides pharmaceutical formulations comprising d4-domperidone of the formula (I): (I) or a pharmaceutically acceptable salt thereof. The formulations also contain (i) a glyceryl stearate, and a medium chain triglyceride; or (ii) a stearoyl polyoxyl glyceride, a nonionic poly(ethylene oxide) polymer, and a medium chain triglyceride; or (iii) a nonionic poly(ethylene oxide) polymer, and a polyethylene glycol. The disclosure also provides methods for treating a disorder that is gastroparesis, nausea apart from gastroparesis, vomiting apart from gastroparesis, nausea associated with gastroparesis, vomiting associated with gastroparesis, gastroesophageal reflux disease, insufficient lactation, or a combination thereof in a patient, comprising administering to the patient a formulation described herein. In some aspects, the disorder is gastroparesis. In other aspects, the disorder is gastroesophageal reflux disease. In further aspects, the disorder is insufficient lactation.
Rescue Group participants aged 21-25 will be connected to a diabetes healthcare "home" (medical clinic or doctor's office) in Los Angeles County based on geography and personal preference. Those individuals connected to the LAC+USC Diabetes Transition Clinic will receive the full 1-year Transition Empowerment Program - Rescue Group" (TEP-RG). Those assigned to other providers in LA County will have access to the web-based curriculum.
BACKGROUND: Predicting complete closure of large ventral hernias without component separation (CS) could have clinical value. The utility of multidetector computed tomography (MDCT)-based component separation index (CSI) was derived and evaluated for these hernias.
METHODS: In 60 patients with a ventral hernia, a CSI was calculated based on their MDCT. In group I (first 30 patients), hernia repair was performed by an open approach and operative assessment determined the need for CS to effect complete closure. A CSI value above which defect closure needed CS was taken as the CSI reference point. In group II (second 30 patients), the hernia repair was done laparoscopically. Patients with a CSI at or below the reference point of group I underwent intraperitoneal onlay mesh (IPOM) after direct closure of the defect (IPOM-plus). The other patients had bilateral endoscopic component separation (ECS) before entry into the peritoneal cavity for an intended IPOM-plus.
RESULTS: A CSI above 0.067 and 0.044 in open and laparoscopic approaches respectively required CS for complete defect closure. A CSI above 0.25 and 0.125 in open and laparoscopic approaches respectively, despite CS, predicted complete closure of defect was not possible.
CONCLUSION: CSI is a more comprehensive parameter for evaluation of ventral hernia than the conventional twodimensional parameters and can predict the need of component separation prior to complete closure of the defect in both laparoscopic and open approach.
Potential candidates for this trial must have received intravenous bisphosphonate therapy (i.e. pamidronate or zoledronic acid) or subcutaneous denosumab for at least 3 months. Baseline patient characteristics will be recorded and screening serum PTH (to rule out primary hyperparathyroidism) and vitamin D (25OH-vit D) will be recorded. In order to fulfill the study objectives, serum will be taken for CTX (fasting morning blood sample), P1NP and BSAP as well as for the optional translational studies (e.g. MMP and TIMP levels). Optional urine will be taken for NTX. Questionnaires will also be completed for symptoms (Brief pain inventory (BPI) \[26\] and FACT-BP \[27\]) and analgesic use (converted into an oral Morphine-equivalent dose). The serum, urine and questionnaire data will be collected at: baseline, weeks 4, 8, and 12. In addition, toxicity information and questions about the occurrence of skeletal related events will be performed at baseline, weeks 4, 8 and 12. At baseline and week 12 the patient will also have a bone marrow aspirate and trephine biopsy performed from the posterior iliac crest. These specimens will be used for ER, PR and Her2 analysis (by FISH) and markers of apoptosis (TUNEL) and proliferation (Ki67) and also for optional translational studies providing tumour cells are present.
The need for continuous anticoagulation represents a potential drawback of RRT modalities. KDIGO 2012 guidelines on acute kidney injury (AKI) suggest the adoption of RCA as first choice anticoagulation modality for continuous RRT (CRRT) in patients without contraindications for citrate. Citrate has been also introduced as anticoagulant for SLED and its use in the context of a mainly diffusive prolonged intermittent modality has been shown to represent an easy and safe method to maintain the extracorporeal circuit when concentrated citrate solutions, such as ACD-A are used. Hypophosphatemia is a known issue of RRT, and has been reported in up to 50-80% of cases when standard RRT solutions are used, especially in continuous/prolonged intermitted modalities when high RRT doses are delivered. RRT-related phosphate depletion should be avoided in critically ill patients due to the relevant hypophosphatemia-associated complications in this clinical setting. The adoption of phosphate-containing CRRT solutions could reduce the incidence of hypophosphatemia and minimize the need for parenteral phosphorus supplementation.The adoption of a commercially available phosphate-containing CRRT solution, in the setting of RCA-RRT may allow to meet the double target of minimizing RRT-induced hypophosphatemia and ensuring an adequate circuit life, also avoiding electrolyte and acid-base derangements.
Clinically significant liver injury has occurred in patients treated with fingolimod in the postmarketing setting. Signs of liver injury, including markedly elevated serum hepatic enzymes and elevated total bilirubin, have occurred as early as ten days after the first dose and have also been reported after prolonged use. Cases of acute liver failure requiring liver transplant have been reported.

In 2-year placebo-controlled clinical trials in adult patients, elevation of liver enzymes (ALT, AST and GGT) to 3-fold the upper limit of normal (ULN) or greater occurred in 14% of patients treated with fingolimod 0.5 mg and 3% of patients on placebo. Elevations 5-fold the ULN or greater occurred in 4.5% of patients on fingolimod and 1% of patients on placebo. The majority of elevations occurred within 6 to 9 months. In clinical trials, fingolimod was discontinued if the elevation exceeded 5 times the ULN. Serum transaminase levels returned to normal within approximately 2 months after discontinuation of fingolimod. Recurrence of liver transaminase elevations occurred with rechallenge in some patients.

Prior to starting treatment with fingolimod (within 6 months), obtain serum transaminases (ALT and AST) and total bilirubin levels. Obtain transaminase levels and total bilirubin levels periodically until two months after fingolimod discontinuation.

Patients should be monitored for signs and symptoms of any hepatic injury. Measure liver transaminase and bilirubin levels promptly in patients who report symptoms that may indicate liver injury, including new or worsening fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. In this clinical context, if the patient is found to have an alanine aminotransferase (ALT) greater than three times the reference range with serum total bilirubin greater than two times the reference range, treatment with fingolimod should be interrupted. Treatment should not be resumed if a plausible alternative etiology for the signs and symptoms cannot be established, because these patients are at risk for severe drug-induced liver injury.

Because fingolimod exposure is doubled in patients with severe hepatic impairment, these patients should be closely monitored, as the risk of adverse reactions is greater [see Use in Specific Populations (8.6), Clinical Pharmacology (12.3)].
The clinical severity of β-hemoglobinopathies is alleviated by the co-inheritance of genetic mutations causing a sustained fetal γ-globin chain production at adult age, a condition termed hereditary persistence of fetal hemoglobin (HPFH). Here, the inventors have compared the extent of fetal hemoglobin (HbF) de-repression following CRISPR/Cas9-mediated targeting of different regions of the HBG1 and HBG2 promoters in an adult erythroid cell line (HUDEP-2). They achieved a potent and pancellular HbF re-activation upon disruption of binding sites for γ-globin repressors located in both HBG1 and HBG2 genes. They validated these findings in Red Blood Cells (RBCs) derived from genome edited Sickle Cell Disease (SCD) patient hematopoietic stem/progenitor cells. Overall, this study identified a binding site for an HbF repressor as a novel and potent target for the treatment of β-hemoglobinopathies. Accordingly, the present invention relates to a method for increasing fetal hemoglobin content in a eukaryotic cell comprising the step of disrupting the binding site for Leukemia/lymphoma-related factor (LRF) in the HBG1 or HBG2 promoter.
AIMS: The REGENERATE-COBRA trial (NCT05711849) will assess the safety and efficacy of an intracoronary infusion of autologous bone marrow-derived mononuclear cells in refractory angina patients with no revascularization options who are symptomatic despite optimal medical and device therapy.
METHODS: REGENERATE-COBRA is a single site, blinded, randomized, sham-controlled, Phase II clinical trial enrolling 110 refractory angina patients with no revascularization options who are symptomatic despite optimal medical and device therapy. Patients will be randomized to either autologous bone marrow derived-mononuclear cells or a sham procedure. Patients in the cell-treated arm will undergo a bone marrow aspiration and an intracoronary infusion of autologous bone marrow derived-mononuclear cells. Patients in the control arm will undergo a sham bone marrow aspiration and a sham intracoronary infusion. The trial's primary endpoint is an improvement in Canadian Cardiovascular Society (CCS) angina class by 2 classes between baseline and 6 months. Secondary endpoints include change in: CCS class at 12 months, myocardial ischemic burden (as measured by perfusion imaging) at 6 months, quality of life at 6 and 12 months (as measured by EQ-5D-5L, EQ-5D-VAS and Seattle Angina Questionnaire), angina frequency at 6 and 12 months, total exercise time (as measured by a modified Bruce protocol) and major adverse cardiovascular events at 6 and 12 months.
CONCLUSIONS: This is the first trial to assess the safety and efficacy of an intracoronary infusion of autologous bone marrow-derived unfractionated mononuclear cells in symptomatic refractory angina patients who have exhausted conventional therapeutic options.
PURPOSE: To obtain a cosmetic, having high humectant effects with persistence and effective in caring for the skin or hair. CONSTITUTION: This cosmetic contains 1-50wt.% phosphoric triester of the formula (R<1> is a 12-24C alkyl or alkenyl; R<2> is a 6-24C alkyl, alkenyl or aryl; R<3> is a 1-6C alkyl, alkenyl or aryl) and 2-90wt.% polyol-based humectant (e.g. dipropylene glycol). The mixing ratio of the compound of the formula to the polyol-based humectant is preferably about 1/2. Furthermore, both the ingredients are blended with a hydrophilic humectant ingredient having >=10wt.% solubility in the polyol-based humectant at 20 deg.C (e.g. dipropylene glycol) and an optional ingredient such as an oil, a surfactant, an ultraviolet light absorber, a humectant or a chelating agent and prepared into an oily, an emulsified or a water-based cosmetic. The cosmetic is especially suitable as a skin or a hair cosmetic requiring humectant effects such as caring for rough skin, hair, etc.
Sepsis is a life-threatening condition characterized by a systemic inflammatory response to infection. Despite extensive research on its pathophysiology, effective therapeutic approaches remain a challenge. This study investigated the potential of resveratrol (RV) and silver nanoparticle-enhanced resveratrol (AgNP-RV) as treatments for sepsis-induced lung injury using a rat model of polymicrobial sepsis induced by cecal ligation and puncture (CLP). The study focused on evaluating changes in oxidative status (TAS, TOS, and OSI) and the expression of inflammatory and apoptotic markers (IL-1β, TNF-α, P2X7R, TLR4, Caspase-3, and Bcl-2) in lung tissue. Both RV and AgNP-RV demonstrated potential in mitigating oxidative stress, inflammation, and apoptosis, with AgNP-RV exhibiting greater efficacy than RV alone (p < 0.05). These findings were corroborated by histopathological analyses, which revealed reduced tissue damage in the RV- and AgNP-RV-treated groups. Our study highlights the therapeutic potential of RV and, particularly, AgNP-RV in combating sepsis-induced oxidative stress, inflammation, and apoptosis. It also underscores the promise of nanoparticle technology in enhancing therapeutic outcomes. However, further investigations are warranted to fully understand the mechanisms of action, especially concerning the role of the P2X7 receptor in the observed effects. Nonetheless, our research suggests that RV and AgNP-RV hold promise as novel strategies for sepsis management.