Objective To analyze the correlation between serum cystatin C (Cys-C) and bone turnover markers in elderly patients with type 2 diabetes mellitus (T2DM) and osteoporosis. Methods A retrospective analysis was conducted on the data of 320 elderly patients with T2DM admitted to Huai'an First People's Hospital from August 2021 to June 2024. Patients were divided into the osteoporosis group and the non-osteoporosis group according to whether they had osteoporosis. General information, bone turnover markers, and serum Cys-C levels were collected from all patients. The data were compared between the two groups to analyze the influencing factors of osteoporosis in elderly patients with T2DM and the correlation between serum Cys-C and bone turnover markers. Results The levels of total cholesterol and LDL-C in the osteoporosis group were higher than those in the non-osteoporosis group, and the bone mineral density was lower than that in the non-osteoporosis group (P<0.05). The levels of TPINP, β-CTX, and Cys-C in the osteoporosis group were higher than those in the non-osteoporosis group, and 25-OH-D3 level was lower than that in the non-osteoporosis group (P<0.05). Serum Cys-C was positively correlated with TPINP and β-CTX, and negatively correlated with 25-OH-D₃ (P<0.05). Multivariate logistic regression analysis found that total cholesterol, LDL-C, TPINP, β-CTX, 25-OH-D₃, and Cys-C were factors influencing osteoporosis in elderly patients with T2DM (P<0.05). Conclusion Serum Cys-C levels in elderly patients with T2DM and osteoporosis are elevated. There is a significant correlation between Cys-C level and bone turnover markers.

ObjectiveTo analyze the adverse reactions associated with the clinical use of Banxia （Rhizoma Pinelliae）- Wutou （Aconitum） in the same formula， with the aim of providing a reference for the safety of their clinical application. MethodsLiterature on the clinical application of antagonistic herbs "Banxia-Wutou" used in the same formula， published from January 1st， 2014， to June 30th， 2023， was retrieved from databases including CNKI， VIP， Wanfang， SinoMed， PubMed， Cochrane Library， and Embase. A database was established， and information related to adverse reactions was extracted， including descriptions， classifications， specific manifestations， management and outcomes， patients' primary diseases （western medicine diseases and traditional Chinese medicine diagnoses and syndromes）， and medication information （dosage， ratio， administration routes， and dosage forms）. ResultsA total of 79 researches simultaneously used antagonistic herbs Banxia-Wutou in the same formula and reported associated advers reactions. Gastrointestinal adverse reactions were the most common， with 8 studies reporting management of adverse reactions and 3 studies reporting improvement with no intervention. Among the 11 researches， the adverse reaction relieved to extant， while other 69 researches didn't report the managment of adverse reaction and its prognosis. For the primary disease in western medicine system， chronic bronchitis and chronic obstructive pulmonary disease （COPD） were most common， while gastric pain was the most common symptom in traditional Chinese medicine with spleen and kidney deficiency and spleen stomach cold deficiency being the most frequent syndromes. The most common Banxia dosage was 10 g， while for the Wutou， Fuzi （Radix Aconiti Lateralis Praeparata） was predominant with the highest dose at 15 g. The most frequent herbal combination was Banxia-fuzi， with a 1∶1 ratio. The main administration route was oral， and the primary dosage form was decoction. ConclusionGastrointestinal adverse reactions are the most common in the clinical use of Banxia-Wutou antagonistic herb combinations. Research on the safety of "Banxia-Wutou" combinations should focus on respiratory system diseases and spleen-stomach related conditions.

The most common medications for the treatment of zoonotic toxoplasmosis are pyrimethamine and sulfadiazine, which may cause serious undesirable side effects. Thus, there is an urgent need to develop novel therapeutics. Baicalein (BAI, C₁₅H₁₀O₅) has been shown to perform well against protozoan parasites including Leishmania and Cryptosporidium. In this study, the inhibition efficacy of BAI on Toxoplasma gondii was evaluated using plaque, invasion, and intracellular proliferation assays. BAI effectively inhibited T. gondii (half-maximum inhibitory concentration (IC₅₀)=6.457×10^-5 mol/L), with a reduced invasion rate (33.56%) and intracellular proliferation, and exhibited low cytotoxicity (half-maximum toxicity concentration (TC₅₀)=5.929×10^-4 mol/L). Further investigation using a mouse model shed light on the inhibitory efficacy of BAI against T. gondii, as well as the potential mechanisms underlying its anti-parasitic effects. The survival time of T. gondii-infected ICR mice treated with BAI was remarkably extended, and their parasite burdens in the liver and spleen were greatly reduced compared with those of the negative control group. Histopathological examination of live sections revealed effective therapeutic outcomes in the treatment groups, with no notable pathological alterations observed. Furthermore, alterations in cytokine levels indicated that BAI not only effectively suppressed the growth of T. gondii but also prevented excessive inflammation in mice. Collectively, these findings underscore the significant inhibitory efficacy of BAI against T. gondii, positioning it as a promising alternative therapeutic agent for toxoplasmosis.
Animals ; Toxoplasma/drug effects* ; Flavanones/therapeutic use* ; Mice ; Antiparasitic Agents/therapeutic use* ; Mice, Inbred ICR ; Toxoplasmosis/drug therapy* ; Female

Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the ＂ temporary import＂ pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.

This paper conducts a pooled analysis of information related to orphan drugs approved for marketing in 2024 by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceuticals and Medical Devices Agency (PMDA) of Japan. It systematically sorts out the scope of indications, therapeutic area distribution, and special review and approval pathways of these drugs, as well as their research and development progress and marketing status in China, thus providing a reference for researchers, regulatory authorities, and industry stakeholders in the field of rare disease drugs.

Objective:To explore the curative efficacy and safety of microwave ablation(MWA)combined with percutaneous ethanol injection(PEI)in treating thyroid cystic solid nodule.Methods:A total of 62 patients with thyroid cystic solid nodule,who admitted to The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University from January 2021 to December 2022,were selected,and they were divided into microwave group(n=28,only single MWA)and combination group(n=34,MWA combined with PEI)according to different treatment plans.The reduction rate of the volume of thyroid nodules,indicators of thyroid function,clinical symptom and the score of physical signs,as well as the incidence of perioperative complications,were compared between two groups after treatment.Results:After treatment,the reduction rate of the volume of thyroid nodule was(88.97±6.36)%in the combination group,which was higher than that(88.15±5.69)%in the microwave group,and the differences in those indicators between the two groups were statistically significant(t=2.465,P＜0.05).The differences of the indicators of thyroid function before and after treatment between two groups were not significant(P＞0.05).The clinical symptoms and the scores of physical signs of both groups after treatment were lower than that before treatment(P＞0.05).The incidence of perioperative complications in the combination group was 17.65%,which was lower than 42.68%in the microwave group(P＜0.05),and the difference of that between the two groups was significant(x2=4.736,P＜0.05).Conclusion:The curative efficacy of the combination of MWA and PEI is better than MWA alone,which has better safety.

Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the ＂ temporary import＂ pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.

Objective:To explore the curative efficacy and safety of microwave ablation(MWA)combined with percutaneous ethanol injection(PEI)in treating thyroid cystic solid nodule.Methods:A total of 62 patients with thyroid cystic solid nodule,who admitted to The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University from January 2021 to December 2022,were selected,and they were divided into microwave group(n=28,only single MWA)and combination group(n=34,MWA combined with PEI)according to different treatment plans.The reduction rate of the volume of thyroid nodules,indicators of thyroid function,clinical symptom and the score of physical signs,as well as the incidence of perioperative complications,were compared between two groups after treatment.Results:After treatment,the reduction rate of the volume of thyroid nodule was(88.97±6.36)%in the combination group,which was higher than that(88.15±5.69)%in the microwave group,and the differences in those indicators between the two groups were statistically significant(t=2.465,P＜0.05).The differences of the indicators of thyroid function before and after treatment between two groups were not significant(P＞0.05).The clinical symptoms and the scores of physical signs of both groups after treatment were lower than that before treatment(P＞0.05).The incidence of perioperative complications in the combination group was 17.65%,which was lower than 42.68%in the microwave group(P＜0.05),and the difference of that between the two groups was significant(x2=4.736,P＜0.05).Conclusion:The curative efficacy of the combination of MWA and PEI is better than MWA alone,which has better safety.

Objective This study aims to sort out the rare disease drugs in the China's Second List of Rare Diseases,to provide reference for the management of rare disease drug treatment.Methods Up to December 31,2023,based on the China's Second List of Rare Diseases,we sorted out the drugs approved in China with the drug label,or approved by the U.S.Food and Drug Administration(FDA)and the European Medicines Agency(EMA)for the treatment of the above diseases,and developed the second batch of rare dis-ease drug catalog in China.The accessibility,localization and coverage of the national medical insurance were also analyzed.Results From the point of view of diseases,a total of 37 diseases in the China's Second List of Rare Diseases have drug indications approved in China,and 10 diseases have drugs listed by the U.S.FDA/EMA and approved in China,but for off-label use.From the point of view of drugs,there are 55 drug indications ap-proved for the treatment of the China's Second List of Rare Diseases,and 22 drugs listed in the U.S.FDA/EMA and approved in the China,but for off-label use.Among the above-mentioned drugs with domestic approved rare disease indications or approved numbers,39 drugs have at least one domestic approval number for a dosage form,covering 30 rare diseases;37 drugs used for at least one rare disease are included in the national medical insur-ance catalog and are covered by reimbursement,covering 29 rare diseases.Conclusions The list of rare disease drugs in the China's Second List of Rare Diseases was established.The number of rare disease drugs and covered diseases approved by China and the U.S.FDA/EMA has increased,and the number of rare disease drugs and covered diseases that are localized and included in the medical insurance catalog has also continued to increase.

Objective To summarize and analyze what the listing in market of orphan drugs in China,the United States,the European Union,and Japan in 2023,and to provide empirical reference to Chinese phar-maceutical enterprises,pharmaceutical management,and new drug research and development departments.Methods Collect the 2023 drug market data released by official drug regulatory agencies in four countries and international organization-China,the United States,the European Union,and Japan categorize the market of rare disease drugs and and their indications,approval dates,R&D status in China,component therapeutic are-as,and special review and approval pathways were analyzed.Results Findings in 2023,28 orphan drugs were listed in the United States,mostly anti-tumor related,accounting for 32.1％(9 out of 28);17 orphan drugs were listed in the European Union,anti-tumor related accounting for 47.0％(8 of 17);22 listed in Japan;and 45 listed in China.In 2023,over 70％orphan drugs listed in the European Union and Japan entered the phase of clinical trials/marketing and applications/approved clinical applications in China(86.4％from Japan and 70.6％from European Union).Conclusions Currently,there is no qualification accreditation mechanism for orphan drugs in China.However,in 2023,the number of rare disease drugs listed in China was the greatest among the four countries/international organization.It reflects that China's Catalog for Rare Diseases played a guiding role in drug research and development.Meanwhile,the reform of drug approval and review in China and the issue of rare disease catalogs took place in a short period of time,so the review and approval,as well as re-search and development of rare disease drugs are still in the catching up stage.