In November 2025, the US Food and Drug Administration (FDA) officially approved Kygevvi oral solution powder for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and children with onset age ≤ 12 years old. Kygevvi is the first and only targeted therapy approved for the treatment of TK2d, consisting of two types of pyrimidine nucleosides, doxecitine and doxribtimine. Its function is to integrate doxecitine and doxribtimine into skeletal muscle mitochondrial deoxyribonucleic acid (DNA), thereby bypassing defective thymidine kinase 2 (TK2) and helping to repair and increase the quantity of mitochondrial DNA, and fundamentally improving cellular energy supply. Here is an introduction to its mechanism of action, pharmacodynamics, pharmacokinetics, clinical research, and safety.

Enhancing the effectiveness of drug regulation and optimizing resource allocation represent core strategies for advancing drug regulatory modernization. To evaluate the efficiency of drug safety regulation in China during the “14th Five-Year Plan” period (2021–2024), analyze the alignment between regulatory resource allocation and output outcomes, identify trends in efficiency changes and regional disparities, and provide recommendations for optimizing the drug regulatory system by bridging the achievements of the “14th Five-Year Plan” with the developmental needs of the “15th Five-Year Plan”.

Data on drug safety regulatory inputs and outputs from 2021 to 2024 were collected. The super-efficiency SBM (slack-based measure) model and Malmquist index were used to measure and analyze drug safety regulatory efficiency across nine provinces in seven administrative regions.

Significant interprovincial efficiency variations were observed. Liaoning maintained consistently high efficiency, Guangdong showed steady improvement, while Shanghai remained persistently low. Slack variable analysis revealed notable input redundancy and output insufficiency in Beijing and Shanghai. Dynamically, only Guangdong, Guangxi, and Shanxi achieved positive growth in total factor productivity (TFP), primarily driven by technological progress (TC>1). In contrast, regions such as Hunan and Guizhou experienced TFP decline due to decreased technical efficiency (EC<1), highlighting significant regional and technological imbalances in the process of improving regulatory effectiveness.

During the “14th Five-Year Plan” period, the efficiency of drug safety regulation in China has seen overall improvement compared to the “13th Five-Year Plan” period. However, challenges such as regional imbalances, lagging pure technical efficiency, and diminishing returns on scale allocation remain. It is essential to advance the transformation of the drug regulatory system toward precision and intelligence, strengthen the development of regulatory talent, and ensure the alignment of regulatory resources with industrial distribution.

To observe the clinical efficacy and safety of aripiprazole tablets combined with magnesium valproate sustained-release tablets in the treatment of patients with bipolar disorder (BD).

BD patients were divided into control group and treatment group according to treatment regimen. The control group was given oral magnesium valproate sustained-release tablets with an initial dose of 0.25 g per time, bid. After 1-2 weeks, the dose was adjusted to 0.75-1.5 g·d^-1 according to the patient’s condition. On the basis of the treatment for the control group, the treatment group was additionally given oral aripiprazole tablets, 5 mg per time, bid. After 2 weeks of medication, the dose was appropriately adjusted to 10 mg per time, bid, according to the patient’s tolerance. Both groups received treatment for 8 weeks. The efficacy, clinical symptom scores, cognitive function, sleep quality, 5-hydroxytryptamine(5-HT), quality of life and adverse drug reactions were compared between the two groups.

A total of 92 cases were enrolled, including 44 in the control group and 48 in the treatment group. After treatment, the clinical efficacy rates in the control group and treatment group were 72.73% (32 cases/44 cases) and 91.67% (44 cases/48 cases), respectively; the Hamilton depression rating scale (HAMD) scores were (7.07±1.38) and (6.38±1.35) points, respectively; the Bipolar disorder rating scale (BRMS) scores were (8.68±2.01) and (7.65±1.16) points, respectively; the positive and negative syndrome scale (PANSS) scores were (52.07±8.97) and (45.27±8.12) points, respectively; the Wisconsin card sorting test (WCST) scores were (51.57±5.66) and (54.13±5.43) points, respectively; the Hopkins Verbal learning test-revised (HVLT-R) scores were (26.27±3.64) and (28.02±3.50) points, respectively; the continuous performance test (CPT) scores were (1.86±0.63) and (2.13±0.57) points, respectively; sleep efficiency rates were (85.45±2.48)% and (89.17±1.17)%, respectively; wakefulness frequency were (3.39±0.81) and (2.92±0.77) times, respectively; 5-HT levels were (52.29±8.23) and (58.09±8.06) ng·L^-1, respectively. Comparisons between the two groups for all the aforementioned indicators showed statistically significant differences (P<0.05, P<0.01, P<0.001). The overall adverse drug reaction rates in the control group and treatment group were 11.36% (5 cases/44 cases) and 14.58% (7 cases/48 cases), respectively (P>0.05).

The combination therapy regimen of aripiprazole tablets and magnesium valproate sustained-release tablets can effectively alleviate clinical symptoms in patients with BD, improve cognitive function and sleep quality, enhance overall quality of life and demonstrate good safety profiles.

To observe the clinical efficacy and safety of budesonide/glycopyrrolate bromide/formoterol fumarate inhalation aerosol combined with theophylline sustained-release tablets in patients with severe chronic obstructive pulmonary disease (COPD) complicated by bronchiectasis (BE).

Patients with BE complicated with severe COPD admitted to our hospital were divided into control group and treatment group based on the treatment method. The control group received theophylline sustained-release tablets (oral, twice daily, 0.1 g per dose), while the treatment group received budesonide/glycopyrrolate/formoterol fumarate aerosol (oral inhalation, twice daily, two puffs per dose) combined with theophylline sustained-release tablets (oral, twice daily, 0.1 g per dose). Both groups were treated for 12 weeks. Inflammatory factor levels, pulmonary ventilation function indicators and clinical efficacy were compared between the two groups, and safety was evaluated.

A total of 133 patients were enrolled, including 71 in treatment group and 62 in control group. Propensity score matching (PSM) was performed at 1∶1 ratio, resulting in 50 patients in each group. After treatment, the total clinical effective rates in control group and treatment group were 80.00% (40 cases/50 cases) and 92.00% (46 cases /50 cases), respectively, with statistically significant difference (P<0.05). After treatment, the levels of C-reactive protein (CRP) in treatment group and control group were (13.85±2.43) and (27.54±3.71) mg·L^-1, respectively; tumor necrosis factor-alpha (TNF-α) levels were (50.54±4.28) and (59.37±4.61) pg·mL^-1, respectively; interleukin-6 (IL-6) levels were (42.30±4.71) and (56.42±5.24) pg·mL^-1, respectively; interleukin-8 (IL-8) levels were (15.71±2.63) and (17.88±3.11) pg·mL^-1, respectively; interleukin-18 (IL-18) levels were (59.42±6.25) and (78.62±7.09) pg·mL^-1, respectively. Differences in the above indicators between the two groups were statistically significant (all P<0.05). After treatment, the forced expiratory volume in 1 second (FEV₁) in treatment group and control group were (2.64±0.37) and (2.06±0.32) L, respectively; forced vital capacity (FVC) were (2.82±0.30) and (2.04±0.27) L, respectively; FEV₁/FVC were (65.68±5.94)% and (54.83±5.55)%, respectively. Differences in the above indicators between the two groups were statistically significant (all P<0.05). The main adverse drug reactions in treatment group were nausea, headache, rash and gastrointestinal discomfort; those in control group were nausea, insomnia, gastrointestinal discomfort and rash. The total incidence of adverse drug reactions was 10.00% in treatment group and 8.00% in control group, with no statistically significant difference (P>0.05).

For patients with BE combined with severe COPD, the regimen of budesonide/glycopyrronium bromide/formoterol fumarate inhalation aerosol combined with theophylline sustained-release tablets demonstrates favorable clinical advantages. It can effectively reduce inflammatory factors in patients and improve their ventilation function.

To investigate the clinical efficacy and safety of atorvastatin calcium tablets in patients with type 2 diabetes cardiomyopathy.

Patients with type 2 diabetic cardiomyopathy admitted to our hospital were divided into treatment group and control group according to the treatment methods. The control group received conventional therapy, while the treatment group was administered atorvastatin calcium tablets at 20 mg·d^-1, orally for 4 weeks. The heart function, blood lipids, blood glucose, autophagy-related proteins and the relative expression levels of phosphatidylinositol 3-kinase/protein kinase B/mammalian target of rapamycin (PI3K/Akt/mTOR) pathway-related mRNA were compared between the two groups, along with a safety evaluation.

A total of 198 patients were included, with 104 in treatment group and 94 in control group. After 4 weeks of treatment, the total clinical effective rate of treatment group was 92.31% (96 cases/104 cases), significantly higher than control group’s 80.85% (76 cases/94 cases) (P<0.05). After treatment, the left ventricular end diastolic diameter of treatment group and control group were (54.41±3.87) and (58.62±3.44) mm, respectively; the left ventricular end diastolic volume were (115.63±8.06) and (121.53±9.88) mL, respectively; the left ventricular end systolic diameter were (30.34±3.52) and (34.84±3.46) mm, respectively; the left ventricular end systolic volume were (69.72±6.32) and (76.57±6.24) mL, respectively; the left ventricular ejection fraction were (52.30±5.53)% and (48.52±4.21)%, respectively; the total cholesterol were (4.48±1.32) and (5.92±1.76) mmol·L^-1, respectively; triglycerides were (1.75±0.52) and (2.38±0.57) mmol·L^-1, respectively; low-density lipoprotein cholesterol were (2.49±0.65) and (3.32±1.21) mmol·L^-1, respectively; high-density lipoprotein cholesterol were (1.02±0.26) and (0.80±0.21) mmol·L^-1, respectively; the levels of benzyl chloride 1 were (3.28±0.43) and (2.62±0.41) ng·mL^-1, respectively; the levels of autophagy related gene 7 were (6.38±1.07) and (4.60±0.86) ng· mL^-1, respectively; the relative expression levels of PI3K mRNA were 0.68±0.25 and 0.94±0.28, respectively; the relative expression levels of Akt mRNA were 0.58±0.27 and 0.95±0.30, respectively; the relative expression levels of mTOR mRNA were 0.66±0.31 and 0.98±0.32, respectively. The above indicators in treatment group were statistically significant compared with control group (all P<0.05). The adverse drug reactions in treatment group include liver dysfunction, gastrointestinal reactions and muscle pain; the control group had abnormal liver function and gastrointestinal reactions. The total incidence of adverse drug reactions in treatment group was 5.77% (6 cases/104 cases), while in control group was 3.19% (3 cases/94 cases). There was no statistically significant difference between the two groups (all P>0.05).

Atorvastatin tablets can significantly improve cardiac structure and function, and regulate blood lipid levels in patients with type 2 diabetic cardiomyopathy. Additionally, it exerts cardioprotective effects by promoting autophagosome formation and downregulating the activity of the PI3K/Akt/mTOR pathway.

To observe the clinical efficacy and safety of tocilizumab injection combined with iguratimod tablets in the treatment of elderly patients with rheumatoid arthritis (RA) complicated with interstitial pneumonia.

Elderly patients with RA complicated with interstitial pneumonia in our hospital were divided into control group and treatment group according to the treatment methods. The control group was given iguratimod tablets 25 mg bid, orally after breakfast and dinner, for a total of 12 weeks on the basis of the conventional treatment plan. The treatment group was given tocilizumab injection 8 mg·kg^-1 intravenously every 4 weeks on the basis of control group. When abnormal liver enzymes, decreased neutrophil count, or decreased platelet count occurred, the dose of tocilizumab injection was reduced to 4 mg·kg^-1, for a total of 12 weeks. The clinical efficacy, lung function and inflammatory response indicators of the two groups were compared, and the safety was evaluated.

A total of 84 cases were included in this study, with 39 cases in control group and 45 cases in treatment group. After treatment, the overall clinical response rates were 74.36% (29 cases /39 cases) in control group and 91.11% (41 cases /45 cases) in treatment group, respectively. The overall clinical response rate of treatment group was statistically significant higher than control group (P<0.05). After treatment, the levels of vital capacity (VC) in control group and treatment group were (2.25±0.34) and (2.38±0.30) L, respectively; the levels of forced expiratory volume in 1 second (FEV₁) were (2.71±0.39) and (2.85±0.36) L, respectively; the high-resolution CT (HRCT) scores were (1.59±0.68) and (1.49±0.59) points, respectively. There was no statistically significant difference in the above indicators between the two groups (all P>0.05). After treatment, the levels of interleukin-6 (IL-6) in control group and treatment group were (13.27±1.95) and (12.05±1.72) pg·mL^-1, respectively; the levels of rapid C-reactive protein (CRP) were (16.09±2.44) and (14.83±2.15) mg·L^-1, respectively; the levels of matrix metalloproteinase-3 (MMP-3) were (60.32±9.59) and (55.61±8.27) μg·mL^-1, respectively; the levels of tissue inhibitor of metalloproteinase-1 (TIMP-1) were (86.79±14.58) and (79.83±13.27) pg·mL^-1, respectively. All the aforementioned indicators in treatment group were statistically significant lower than control group (P<0.05, P<0.01). The main adverse drug reactions in control group were elevated aminotransferase, leukopenia and upper abdominal pain. The main adverse drug reactions in treatment group were elevated aminotransferase, leukopenia, decreased neutrophil count and upper abdominal pain. The total incidence of adverse drug reactions was 7.69% in control group and 11.11% in treatment group, with no statistically significant difference (P>0.05).

Tocilizumab injection combined with iguratimod tablets is effective in the treatment of elderly patients with RA complicated with interstitial pneumonia, and can effectively improve their clinical symptoms, serum marker levels, and lung function, reduce inflammatory responses, and has good safety.