Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures, vosoritide has demonstrated favorable efficacy and a reassuring safety profile. Nevertheless, existing international ACH guidelines largely emphasize complication management and symptomatic care, and there is no unified consensus on pharmacologic therapy. To address this gap, an international expert group developed the International Consensus Guidelines for the Implementation and Monitoring of Vosoritide Therapy in Patients with Achondroplasia providing systematic recommendations that span the continuum of care - from initial patient contact and pre-treatment assessment to medication counseling, injection training, and long-term outcome monitoring. These recommendations complement and refine current management and nursing protocols for individuals with ACH and offer practical guidance for clinicians across diverse regions. This article highlights key elements of the guideline to provide evidence-based support and clinical direction for healthcare professionals in China treating children with ACH using vosoritide.
Humans ; Achondroplasia/drug therapy* ; Consensus ; Practice Guidelines as Topic ; Child

AIM: To investigate the value of maximum standardized uptake value(SUVmax), extraocular muscle(EOM)diameter, and thyroid stimulating hormone receptor antibody(TRAb)in evaluating inflammatory activity in thyroid-associated ophthalmopathy(TAO).METHODS: TAO patients underwent orbital 99mTc-DTPA SPECT/CT imaging at the Affiliated Hospital of Panzhihua University between January 2023 and December 2024 were retrospectively enrolled. According to the clinical activity score(CAS), patients were classified into an active group and an inactive group. SUVmax and EOM were independently measured by three nuclear medicine physicians, and the mean values were used for analysis. Serum TRAb levels from TAO patients were measured using a fully automated electrochemiluminescence immunoassay analyzer. Nonparametric tests were used to compare differences between the two groups. Spearman correlation analysis was performed to assess the correlations of CAS with SUVmax, EOM, and TRAb. Using CAS as the reference standard, receiver operating characteristic(ROC)curves were plotted to evaluate the diagnostic performance of SUVmax, EOM, and TRAb, as well as their combination. Intraclass correlation coefficient(ICC)was used for reliability analysis to evaluate the consistency of SUVmax and EOM among the 3 measurers.RESULTS: A total of 99 patients(198 eyes)with TAO were enrolled in this study, including 38 males and 61 females, aged 16-74(46.92±11.84)y. The active group comprised 46 patients(92 eyes), aged 16-71(48.40±12.02)y, with 25 males and 21 females. The inactive group included 53 patients(106 eyes), aged 17-74(45.46±11.59)y, consisting of 13 males and 40 females. There were no statistically significant differences between the two groups in terms of gender and age(P>0.05). Statistically significant differences were observed in SUVmax, EOM, and TRAb. Compared with the inactive group, the active group showed higher SUVmax, EOM, and TRAb levels(all P<0.01). Correlation analysis showed that CAS was positively correlated with SUVmax, EOM, and TRAb(r=0.734, 0.622, and 0.404, respectively, all P<0.01). ROC curves were plotted using CAS as the reference standard, and the AUCs for SUVmax, EOM, TRAb, and their combined diagnosis were 0.874, 0.797, 0.715, and 0.892, respectively. The ICC of SUVmax and EOM were 0.925(95%CI: 0.901-0.948, P<0.001)and 0.943(95%CI: 0.907-0.964, P<0.001)respectively.CONCLUSION: Beyond CAS, there is a need to identify objective imaging and serological biomarkers. Measurements of SUVmax and EOM provide visualized and quantifiable tools for assessing TAO activity, and their combination with TRAb can further improve the accuracy of activity assessment.

Sichuan Province is a hilly schistosomiasis-endemic province in China with the highest endemicity and largest coverage. During the period of more than 70 years of integrated control, there are five distinct phases of schistosomiasis control in Sichuan Province, including baseline investigation, Oncomelania hupensis snail control, chemotherapy for both humans and livestock, management of source of Schistosoma japonicum infections, and sustained control and intensified surveillance and early warning, and a package of key interventions are implemented, including environmental modification, snail control with chemical treatment, large-scale chemotherapy for humans and livestock, management of source of S. japonicum infections, intensified integrated control, establishment of an early warning and surveillance system. As a result, schistosomiasis has been eliminated across all 63 disease-endemic counties (cities, districts) in Sichuan Province by June 2025. Government-led multi-sectoral collaborations, science and technology-supported precision control strategies, and a sustainable control model tailored to the ecological and socio-economic conditions in endemic regions are essential to achieving the target of schistosomiasis elimination in Sichuan Province.

Objective: To analyze the changes in serum ghrelin and leptin levels in girls with central precocious puberty (CPP) after 24 weeks of aerobic training combined with gonadotropin releasing hormone agonist (GnRH-a) therapy, so as to provide reference for preventing girls from precocious puberty. Methods: A total of 96 girls with CPP who were treated at the Affiliated Huai an Hospital of Xuzhou Medical University between April 2022 and January 2024 were selected as study subjects. They were divided into a pharmacotherapy group and a combined pharmacotherapy and aerobic exercise group by random number table method, with 48 patients in each group. The pharmacotherapy group received subcutaneous injections of leuprorelin acetate microspheres at a dose of 100 μg/kg in the morning (8:00-10:00), administered once every 4 weeks for a total of 6 injections. The combined pharmacotherapy and aerobic exercise group participated in aerobic exercise in addition to the subcutaneous injections of leuprorelin acetate microspheres; the training was conducted for 24 weeks, 3 times per week, and was conducted after school for girls, for 20- 75 min per session. The t test was utilized to comparatively analyze the serum leptin and ghrelin levels of the patients in both groups before and after the intervention. Binary Logistic regression analysis was applied to explore the correlative factors influencing serum ghrelin and leptin levels in girls with CPP post intervention. Results: Following the intervention, the serum leptin [(7.85± 1.43)ng/mL ] and ghrelin [(8.46±1.06)ng/mL] levels in the girls of the pharmacotherapy group demonstrated no statistically significant changes compared to their pre intervention levels [(7.94±1.45) (8.35±1.22)ng/mL, respectively] ( t =0.41, -0.58, both P > 0.05 ). Conversely, in the combined pharmacotherapy and aerobic exercise group, the post intervention serum leptin levels [(6.35±1.62) ng/mL] significantly decreased compared to pre intervention [(7.87±1.59) ng/mL], and the serum ghrelin levels [(9.12±1.37) ng/mL] significantly increased compared to pre intervention [(8.22±1.47) ng/mL] ( t =6.20, 4.39, both P <0.05). Conclusion Aerobic training increases serum ghrelin levels and decreases serum leptin levels in girls with CPP.

This paper systematically analyzes schistosomiasis transmission risks during the elimination stage and reviews the evolution of the schistosomiasis surveillance system in China from a One Health perspective, with a special emphasis on schistosomiasis surveillance and response practices during the elimination stage from a One Health perspective. These practices include precise screening of human schistosomiasis under low-endemicity conditions, systematic monitoring of schistosomiasis in domestic and wild animals, ecological management of Oncomelania snails, and information-coordinated responses. Nevertheless, the current surveillance and response system for schistosomiasis suffers from challenges of insufficient cross-sectoral coordination and complex external environments, which constrain precise schistosomiasis surveillance and response in China. Therefore, it is urgently necessary to integrate coordination mechanisms, empower the surveillance system with technological innovation, improve capacity building, and actively promote diverse social participation in the One Health framework, so as to consolidate current schistosomiasis control achievements and achieve the target for schistosomiasis elimination across China, which may provide a replicable technical model for global zoonoses control.

The abnormal accumulation, structural change, and spread of tau protein in the brain are now considered important factors in the progression of Alzheimer’s disease (AD) and many other tau-related neurodegenerative diseases. Clinical and pathological studies have shown that the amount and distribution of abnormal tau protein are closely related to synaptic damage, neuronal loss, and cognitive decline. Therefore, tau protein has become an important target for disease-modifying therapy. However, tau is difficult to target because it has no fixed structure, lacks a clear active site, undergoes many post-translational modifications, and forms different abnormal structures in different diseases and disease stages. Therefore, the effective and selective removal of harmful tau species is both a major opportunity and a major challenge in drug discovery for neurodegenerative diseases. Proteolysis-targeting chimeras (PROTACs) offer a new strategy to deal with this problem. Unlike traditional inhibitors, PROTACs do not simply block protein function. Instead, they bring the target protein close to an E3 ubiquitin ligase, which leads to ubiquitination and degradation of the target protein by the proteasome. This event-driven mechanism may be especially useful for disease-related proteins such as tau protein, which are hard to inhibit with traditional small molecules. In recent years, tau protein-targeting PROTACs and related degradation strategies have shown promising effects in cell and animal models. These effects include reducing abnormal tau protein levels, lowering synaptic toxicity, regulating disease-related signaling pathways, and improving behavioral or cognitive outcomes. These studies support targeted tau protein degradation as a promising therapeutic approach, although many problems still need to be solved before clinical use. In this review, we summarize recent progress in the development of tau protein-targeting PROTACs and discuss the main factors that affect their design and activity. We focus on the choice of tau protein-binding ligands, their binding sites, and their ability to recognize abnormal tau protein rather than normal tau protein. We also discuss the selection of E3 ligase recruiters, linker length and structure, ternary complex formation, and structure-activity relationships that influence degradation potency and selectivity. In addition, we introduce new strategies beyond traditional proteasomal degradation, including the regulation of tau protein phosphorylation and autophagy-based clearance. These approaches may be more suitable for different tau species or different stages of disease. A major challenge for tau protein-targeting PROTACs is delivery to the central nervous system. Many PROTACs have high molecular mass, large polar surface area, flexible structures, and may be removed by efflux transporters. As a result, they often have poor ability to cross the blood-brain barrier and may not reach enough free drug levels in the brain. Therefore, the therapeutic potential of tau protein-targeting PROTACs depends not only on their ability to degrade tau protein in vitro, but also on whether they can achieve long-lasting, selective, and safe target engagement in important brain regions after practical administration. Future studies should focus on improving selectivity for abnormal tau protein, understanding E3 ligase function in neural cells, designing brain-penetrant molecules, developing better delivery methods, and testing these drugs in models that better reflect human disease. Overall, tau protein-targeting PROTACs are a promising and fast-developing strategy for treating tau-related diseases. However, their successful clinical translation will require progress in chemical biology, medicinal chemistry, neuroscience, drug delivery, and translational medicine.

Objective To explore the symptom clusters in renal transplant recipients and con-struct a concurrent symptom network to identify core symptoms.Methods A total of 343 patients with followed up after renal transplantation were selected as the study subjects.A general information questionnaire and the Chinese version of the Modified Transplant Symptom Occurrence and Symptom Distress Scale were employed to analyze the occurrence of symptoms in patients.In this study,only symptoms with an incidence rate greater than 20％and Spearman correlation coefficient greater than 0.40 between symptom severity and total score were retained.Exploratory factor analysis was used to extract symptom clusters with a factor loading of ≥0.45 as the criterion.The R language was utilized to construct symptom network,based on which core symptoms and bridge symptoms were identified.Results A total of 5 symptom clusters were extracted in this study:the neuro-gastrointestinal symp-tom cluster,the mood-related symptom cluster,the hormone-related symptom cluster,the energy de-ficiency symptom cluster and the vision-related symptom cluster.The core symptoms were anxiety(rs=1.75),mood swings(rs=1.50)and muscle weakness(rs=1.27).The top three symptoms in terms of bridge strength were muscle weakness(rb=0.87),lack of vitality(rb=0.66)and fa-tigue(rb=0.65).Conclusion Multiple symptoms are presented in patients after renal transplanta-tion.Based on the results of symptom network analysis,clinicians can strengthen the assessment of core symptoms and bridge symptoms to develop precise intervention strategies and improve the effectiveness of symptom management.

Objective:To study the changes in biliary fluid dynamics in patients with hepatolithiasis after cholecystectomy.Methods:The clinical data of 101 patients with hepatolithiasis who underwent percutaneous transhepatic scleroscopic choledochotomy for stone extraction at the First Hospital of Guangzhou Medical University from September 2021 to June 2024 were retrospectively analyzed, among which there were 47 males and 54 females with the age of (51.8±15.7) years. They were divided into two groups based on whether they had undergone previous cholecystectomy or not: cholecystectomy group ( n=53) and non-chole-cystectomy group ( n=48). The pressures in the left hepatic duct, right hepatic duct and lower end of the common bile duct were compared between the two groups, as well as the viscosity of bile at different rates of incision. Results:There were no significant differences in baseline characteristics such as gender, age, and liver function between the two groups (all P>0.05). Compared with the non-cholecystectomy group, the bile viscosity in the cholecystectomy group were significantly lower at shear rates of 1/s, 50/s, and 200/s [1/s: (8.96±1.15) mPa·s vs. (13.13±1.25) mPa·s; 50/s: (2.37±0.18) mPa·s vs. (3.59±0.34) mPa·s; 200/s: (1.82±0.13) mPa·s vs. (2.25±0.15) mPa·s], with statistically significant diffe-rences (all P<0.05). The biliary pressure in the left hepatic duct, right hepatic duct, and lower end of the common bile duct in the cholecystectomy group were significantly higher than that in the non-cholecystectomy group [left hepatic duct: (16.43±7.02) cmH 2O vs. (13.84±5.07) cmH 2O; right hepatic duct: (16.71±7.36) cmH 2O vs. (13.76±5.03) cmH 2O; lower end of the common bile duct: (14.60±6.73) cmH 2O vs. (10.58±4.84) cmH 2O] (1 cmH 2O=0.098 kPa), with statistically significant differences (all P<0.05). Conclusion:Bile viscosity decreases after cholecystectomy in patients with hepatolithiasis, whereas biliary pressure increases at the left and right hepatic ducts and at the lower end of the common bile duct, and these changes may be closely related to the mechanism of hepatolithiasis formation and recurrence.

AIM To study the chemical constituents from Commelina communis L.METHODS The 95％ethanol extract from C.Communis was isolated and purified by activated charcoal,silica gel,Sephadex LH-20,and HPLC,then the structures of obtained compounds were identified by physicochemical properties and spectral data.RESULTS Seventeen compounds were isolated and identified as p-hydroxyl ethyl cinnamate(1),p-hydroxybenzaldehyde(2),vanillin(3),4-hydroxy-2,3-dimethyl-2-nonen-4-olide(4),hemeratrol A(5),chakyunglupulin B(6),chakyunglupulin A(7),2-(2-hydroxyethyl)-3-methylfumaric acid(8),N-cis-feruloyl tyramine(9),N-trans-coumaroyltyramine(10),5,6,7,3',4',5'-hexamethoxyflavone(11),N-trans-sinapoyltyramine(12),dihydro-feruloyltyramine(13),N-trans-feruloyltyramine(14),2-phenylethanol-β-D-glucoside(15),quercetin-3-O-β-D-glucoside(16),and isorhamnetin-3-O-β-D-glucopyranoside(17).CONCLUSION Compounds 4-8,10 and 11 are isolated from Commelina genus for the first time,and 1,9,12-15 are first isolated from this plant.

Background::An evidence gap still exists regarding the efficacy and safety of tegoprazan in patients with erosive esophagitis (EE) in China. This study aimed to verify the efficacy and safety of tegoprazan vs. esomeprazole in patients with EE in China. Methods::This study was a multicenter, randomized, double-blind, parallel, active-controlled, non-inferiority phase III trial of patients with EE randomized 1:1 to tegoprazan 50 mg/day vs. esomeprazole 40 mg/day. This study was conducted in 32 sites between October 24, 2018 and October 18, 2019. The primary endpoint was the cumulative endoscopic healing rate at week 8. The secondary endpoint included endoscopic healing rate at week 4, changes in the reflux disease questionnaire (RDQ) and gastroesophageal reflux disease health-related quality of life (GERD-HRQL) scores, and symptom improvement. Results::A total of 261 patients were randomized: 132 to the tegoprazan group and 129 to the esomeprazole group. The cumulative endoscopic healing rate at 8 weeks in the tegoprazan group was non-inferior to that of the esomeprazole group (91.1% vs. 92.8%, difference: -1.7%, 95% confidence interval [CI]: -8.5%, 5.0%, P = 0.008). There were no statistically significant differences in the changes in RDQ (total, severity, and frequency) and GERD-HRQL scores between the two groups (all P >0.05). The percentages of days without symptoms, including daytime and nighttime symptoms based on patients' diaries, were similar between the two groups (all P >0.05). In the tegoprazan and esomeprazole groups, 71.5% (93/130) and 61.7% (79/128) of the participants reported adverse events (AEs), 2.3% and 0 experienced serious AEs, while 70.0% and 60.2% had treatment-emergent AEs, respectively. Conclusion::Tegoprazan 50 mg/day demonstrated non-inferior efficacy in healing EE, symptom improvement, and quality of life, and it has similar tolerability compared with esomeprazole 40 mg/day.