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Cystic fibrosis is the most common life-limiting genetic disease in the Caucasian population, with median predicted survival progressively improving up to 50 years, thanks to highly standardized multidisciplinary approach. Patients with p.Phe508del homozygosity usually have poorer lung function and higher mortality rates per year than other groups. By reason of that, this population has been among the most eligible target of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a new class of drugs that can partially restore CFTR function by the correction of CFTR misfolding and trafficking to the cell surface. This narrative review summarizes the current preclinical and clinical evidence of the triple combination of elexacaftor-tezacaftor-ivacaftor, the new benchmark among highly effective CFTR modulators. It provides details on the efficacy and safety that led to drug regulation and approval and discusses future developments in clinical and translational research.

The therapeutic strategies for hepatocellular carcinoma (HCC) have greatly expanded in recent years. However, the actual usage of each of these treatments in clinical routine remains unknown. Here, we analysed the distribution and changes of the main surgical and radiological therapeutic procedures nationwide during the last decade.

Retrospectively, analysis of the data on all >18-year-old patients with a diagnosis of HCC identified in the French Program for the Medicalization of Information Systems database that contains all discharge summaries from all French hospitals. The number and percentage of the therapeutic procedures performed from January 2010 to December 2019 were extracted.

A total of 68,416 therapeutic procedures were performed in 34,000 HCC patients. Whereas HCC incidence remained stable, the annual number of procedures frankly increased over the decade (from 4267 to 8042). Trans-arterial chemoembolization was the most frequently performed technical procedure, with a double-digit annual growth from 2010 (n=1932) to 2015 (n=4085), before stabilization from 2016. Selective internal radiation therapy displayed the highest increase in the decade (+475%). Among curative treatments, the annual number of percutaneous tumour ablations more than doubled in 10 years, till representing 64% of curative treatments in cirrhotic patients in 2019. Surgical tumour resections showed a 1.5-fold increase in 10 years, due to the great increase in minimally invasive approaches, whereas the proportion of open resection progressively decreased.

Minimally invasive procedures have gained major importance in HCC management during the last decade. Percutaneous thermal ablation has emerged as the first curative treatment performed for patients with HCC.

Minimally invasive procedures have gained major importance in HCC management during the last decade. Percutaneous thermal ablation has emerged as the first curative treatment performed for patients with HCC.

In epilepsy stigma, certain perceptions are culturally dependent and greatly influence a person's attitudes. Hence, we aimed to explore the perceptions associated with attitudes toward epilepsy in various urban subpopulations.

This is a mixed-method study employing the Public Attitude Toward Epilepsy (PATE) scale as the quantitative measure, followed by a semi-structured interview. The qualitative data were then counted and analyzed concurrently with the quantitative data.

A total of 410 respondents (104 people with epilepsy [PWE]; 104 family members [FM]; 100 medical students [MS]; 102 public [Pb]) aged 37 years (IQR 23-55) were recruited. They were mostly female (57.3%), Chinese (52.0%), and highly educated (63.7%). The attitudes toward epilepsy among medical students are the best, followed by the PWE and their family members, and the worst among the public. The qualitative results revealed 4 main themes, which were "general social values", "epilepsy severity and control", "PWE's abilities", and "harmvaried between subpopulations, attitude levels, domains, and aspects of life. (304 words).

The perceptions underlying attitudes toward epilepsy were complex and varied between subpopulations, attitude levels, domains, and aspects of life. (304 words).

This study aimed to determine the self-efficacy levels of individuals with epilepsy and also compare individuals' self-efficacy with sociodemographic and epilepsy-related characteristics.

The cross-sectional study was carried out with 200 individuals with epilepsy who were suitable for the inclusion criterias. Data were collected using patient information form which includes sociodemographic and clinical characteristics and Epilepsy Self-Efficacy Scale for evaluating epilepsy-related self-efficacy levels of patients.

The self-efficacy levels of the patients were moderate. Education, gender, occupational status, income, seizure frequency, regular use of medication, the number of drugs used, patient behavior following a drug-related problem, family support, the ability to obtain information about the illness, and hiding the illness from other people were significant factors determining scores on the epilepsy self-efficacy scale.

This study confirms the findings of previous studies that were conducted to determine factors affecting self-efficacy. In order to increase self-efficacy, the findings show that there is a need for social support, education of epilepsy individuals, and information and awareness in society.

This study confirms the findings of previous studies that were conducted to determine factors affecting self-efficacy. In order to increase self-efficacy, the findings show that there is a need for social support, education of epilepsy individuals, and information and awareness in society.18F-Labelled pyrrolopyrimidines were synthesized and evaluated as positron emission tomography (PET) probes to determine leucine-rich repeat kinase 2 (LRRK2) expression in the brain. With pyrrolopyrimidine derivative PF-06447475 as the lead compound, two in vivo-stable 18F-labelled pyrrolopyrimidines ([18F]1 and [18F]2) were synthesized automatically at radiochemical yields 8-10% (non-decay-corrected) with molar activities of 0.95 and 0.5 GBq/μmol, respectively. GSK 2837808A datasheet The measured Kd of 6.90 nM for 1 and 14.27 nM for 2 demonstrated high affinities for LRRK2. The LRRK2 G2019S mice had higher uptakes (P less then 0.01) of [18F]1 in the olfactory bulb, striatum, and hippocampus than WT mice during microPET/CT imaging, consistent with immunohistology results of LRRK2 distribution. [11C]CFT microPET/CT imaging demonstrated a lower expression of dopamine transporter in LRRK2 G2019S mice. Parkinson's disease-like deficits in dopamine transporter synthesis and cognitive declines were noticed along with LRRK2 expression increase in the olfactory bulb, striatum, and hippocampus.

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