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Factor X + Factor IX (Factor X P Behring)

✓ Approved

CSL Behring · F9 · 细胞治疗

什么是 Factor X + Factor IX?

Factor X + Factor IX 是一种细胞治疗,由CSL Behring研发。该药已获批,用于治疗相关适应症,给药途径:Injectable (Others)、Intravenous (IV)。

药物档案

商品名Factor X P Behring
公司CSL Behring
药物类别细胞治疗
分子靶点F9, F10
给药途径Injectable (Others), Intravenous (IV)
状态Approved

作用机制

分子靶点

Factor X + Factor IX 作用于 2 个分子靶点:

F9coagulation factor IX (P19, F9 p22)
F10coagulation factor X (FXA, FX)
需要更深入的分析?Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

Factor X + Factor IX 针对 2 个适应症,涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersFactor IX deficiency✓ Approved
Congenital, familial and genetic disordersFactor X deficiency✓ Approved

相关研究文献

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Ultrasensitive SERS detection of trace malachite green using noble-metal-free EuFeO3/ZnO@Ti3C2T x MXene heterostructures.

Nguyen Thi Thanh Huong TTH, Nguyen Anh Tien AT, Giang Tri Danh TD, Pham Thi Thuy TT et al.

Malachite green (MG), an illegally used aquaculture dye with carcinogenic and mutagenic effects, remains a persistent contaminant in aquatic environments and food chains, necessitating the development of highly sensitive and reliable analytical platforms for trace-level monitoring. In this study, a hierarchical EuFeO3/ZnO@Ti3C2T x MXene heterostructure was successfully engineered as a noble-metal-free surface-enhanced Raman scattering (SERS) substrate for ultrasensitive MG detection in aquaculture water. The hierarchical assembly of EuFeO3 and ZnO on conductive Ti3C2T x MXene generated electronically coupled heterointerfaces that facilitated rapid charge transfer and efficient carrier transport. Structural and spectroscopic analyses confirmed the successful formation of the ternary architecture with intimate interfacial integration, enhanced visible-light absorption, enlarged surface accessibility, and significantly suppressed charge-carrier recombination compared with pristine and binary systems. Consequently, the EuFeO3/ZnO@Ti3C2T x substrate delivered markedly amplified Raman responses, achieving ultrasensitive MG detection at concentrations as low as 10-9 M with an enhancement factor of 7.31 × 106. The substrate further demonstrated excellent linearity, reproducibility, and stable analytical performance in complex aquaculture water matrices, achieving recoveries ranging from 82.7% to 111.8% with relative standard deviations below 13.8%. Mechanistic investigations revealed that the enhanced SERS activity was predominantly governed by chemical enhancement arising from interfacial charge-transfer interactions between the heterostructure and MG molecules, while the conductive MXene scaffold accelerated electron transport and strengthened analyte-substrate coupling. This work not only demonstrates an effective strategy for constructing MXene-based semiconductor heterostructures with enhanced charge-transfer characteristics, but also provides a promising noble-metal-free SERS platform for practical food safety monitoring and environmental contaminant detection.

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PubMedFrontiers in psychology2026-08-05

The cross-cultural adaptation and psychometric evaluation of the Chinese version of the stigma scale for caring for individuals with sexually transmitted infections (STISS)-student version: a translation and validation study.

Shi Jiahao J, Lin Youbei Y, Li Chuang C, Gong Yinghui Y et al.

Stigmatization of patients with sexually transmitted infections remains prominent, whereas previous measurement tools have mostly focused on single-disease populations and lack a systematic assessment of the broader population of individuals with sexually transmitted infections. To translate the Stigma Scale for Caring for Individuals with Sexually Transmitted Infections (STISS) - Student Version and evaluate its reliability and validity among nursing students in China. This study recruited 413 nursing students through convenience sampling based on Kendall's sample size estimation method. The original version of the scale was translated according to the Brislin guidelines, and content validity and the feasibility of the translation were validated through expert consultation. Structural validity was assessed using exploratory factor analysis and confirmatory factor analysis. Reliability was tested using internal consistency reliability. The Chinese version of the STISS scale demonstrated good psychometric properties. The Cronbach's alpha coefficient was 0.921. The four-factor exploratory factor model explained 58.947% of the total variance, indicating a robust factor structure. Confirmatory factor analysis showed the following fit indices: χ 2/df = 1.078; RMSEA = 0.019; CFI = 0.994; TLI = 0.993; GFI = 0.927; AGFI = 0.907. All indices were within acceptable ranges, and both convergent validity and discriminant validity were adequately confirmed. This study strictly followed the Brislin translation model and successfully introduced the STISS scale, which demonstrated strong reliability and validity within the Chinese cultural context. It is highly suitable for measuring the level of stigmatization of individuals with sexually transmitted infections among nursing students in China.

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Bayesian joint additive factor models for multiview learning.

Anceschi Niccolo N, Ferrari Federico F, Dunson David B DB, Mallick Himel H

It is increasingly common to collect data of multiple different types on the same set of samples. Our focus is on studying relationships between such multiview features and responses. A motivating application arises in the context of precision medicine where multiomics data are collected to correlate with clinical outcomes. It is of interest to infer dependence within and across views while combining multimodal information to improve the prediction of outcomes. The signal-to-noise ratio can vary substantially across views, motivating more nuanced statistical tools beyond standard late and early fusion. This challenge comes with the need to preserve interpretability, select features, and obtain accurate uncertainty quantification. To address these challenges, we introduce two complementary factor regression models. A baseline joint factor regression (jfr) captures combined variation across views via a single factor set, and a more nuanced Joint Additive FActor Regression (jafar) that decomposes variation into shared and view-specific components. For JFR, we use independent cumulative shrinkage process (I-CUSP) priors, while for JAFAR, we develop a dependent version (D-CUSP) designed to ensure identifiability of the components. We develop Gibbs samplers that exploit the model structure and accommodate flexible feature and outcome distributions. Prediction of time-to-labor onset from immunome, metabolome, and proteome data illustrates performance gains against state-of-the-art competitors.

PMID 42554212
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Chan Lily Man Lee LML, Choi Edmond Pui Hang EPH, Lam Wendy Wing Tak WWT, Chan Koon Ho KH et al.

Palliative care has been advocated to improve symptom burden and quality of life among people with progressive neurological diseases. However, validated palliative care outcome measures for neurological conditions remain limited in non-Western settings. To translate, adapt and psychometrically evaluate the Integrated Palliative care Outcome Scale for Neurological conditions (IPOS-Neuro) among people with progressive neurological diseases in Hong Kong. The traditional Chinese version of IPOS-Neuro was developed through forward-backward translations, cognitive debriefing interviews, and expert review. Psychometric properties were evaluated by examining factor structure, convergent validity, concurrent validity, internal consistency, and test-retest reliability. Comparator instruments included Palliative Care Outcome Scale, Hospital Anxiety and Depression Scale, and EQ-5D-5L. Two hundred and ten adults with progressive neurological diseases recruited from regional neurology outpatient clinics and patient support groups in Hong Kong. Confirmatory factor analysis supported the three-factor structure (Physical Symptoms, Emotional Symptoms, and Communication/Practical Issues) with acceptable fit indices. IPOS-Neuro showed good internal consistency (Cronbach's alpha = 0.93), strong concurrent validity with Palliative Care Outcome Scale (r = 0.74), moderate convergent validity with EQ-5D-5L index (r = -0.51) and Hospital Anxiety and Depression Scale (r = 0.56), and excellent test-retest reliability (intraclass correlation coefficient = 0.99). Additional exploratory factor analysis identified a new clinically-meaningful nine-factor model: psychosocial problems and symptom dimensions related to fatigue, motor, gastrointestinal, oral and sensory, cognitive, sexual, bowel, and non-motor issues. The traditional Chinese version of IPOS-Neuro showed promising psychometric properties for assessing palliative care needs in progressive neurological diseases. Future validation in larger and diverse neurological disease samples is warranted.

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PubMedGenetics in medicine : official journal of the American College of Medical Genetics2026-08-05

The Patient-reported Genetic testing Utility InDEx (P-GUIDE): A novel measure of personal utility.

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Consensus is lacking on how to define and measure the concept of personal utility in genomic medicine. The study objective was to develop and assess the validity of the Patient-reported Genetic testing Utility InDEx (P-GUIDE) for pediatric genetics. Informed by the literature, experts, and patient partners, a 55-item first draft of P-GUIDE was developed. Interviews were conducted with parents of children who had genetic testing to assess item clarity and relevance. Clinical experts assessed content validity. Two independent sequential samples of parents completed questionnaires. Item reduction was achieved using exploratory factor analysis and confirmatory factor analysis. Performance characteristics were assessed on the final tool. Interviewees (n=22) found most items relevant but requiring modifications. Clinical experts (n=11) recommended further revisions. Exploratory factor analysis (n=103) removed 9 items. Confirmatory factor analysis (n=103) generated a final tool consisting of 19 items across three factors: understanding and decision-making, psychosocial benefit, and psychosocial concern. P-GUIDE scores were positively correlated with published measures of utility, knowledge, empowerment, and emotion, establishing construct validity. P-GUIDE demonstrated strong test-retest reliability. P-GUIDE is available for use with pediatric populations. Emerging adaptations of P-GUIDE contribute to a novel utility measurement system for genetic testing.

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Simulation-based training is crucial for acquiring emergency medicine competencies during medical education. In Germany, teaching is largely competency-based and guided by the National Competence Based Catalogue of Learning Objectives for Undergraduate Medical Education (NKLM) 2.0. Given the diversity of clinical scenarios and varying student expertise, a standardized assessment instrument is required for objective and reproducible performance evaluation. Moreover, such instruments are essential for educational research. We developed a checklist aligned with NKLM 2.0 and established emergency medicine guidelines. Content validity was ensured through expert panel review. The checklist assessed three domains (evaluation, background, and action) corresponding to key elements of the ISBAR framework. The instrument was applied to 265 medical students across different semesters and multiple simulation scenarios using purposive sampling and including all students from two semesters willing to participate. Construct validity was examined using factor analysis. Internal consistency was assessed using Cronbach's α, and interrater reliability by calculating variance attributable to rater and station effects. After exclusion of one item with Kaiser-Meyer-Olkin estimator of 0.45, exploratory and confirmatory factor analyses supported a three-factor structure, with items loading on evaluation (CFA-loadings 0.831-0.901), background (0.582-0.736), and action (0.491-0.759). The CFA showed good model fit (CFI = 1.0, TLLI = 1.014, RMSEA < 0.001, SRMR = 0.045). Factors were moderately correlated (0.735-0.882), suggesting related dimensions of a common construct. Rater and station effects accounted for 14.2% of total variance. Cronbach's α was 0.730 overall, and 0.721 (evaluation), 0.557 (background), and 0.604 (action), respectively. Alignment with NKLM 2.0 supported content validity, while factor analysis confirmed the proposed construct structure. Overall internal consistency and acceptable rater-related variance as well as factor-correlation indicate that the checklist is a valid and reliable instrument for assessing emergency medicine competencies in undergraduate simulation training, though subscore interpretation remains limited due to low internal consistency of individual domains.

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