Donepezil, an acetylcholinesterase inhibitor, is widely used for managing the symptoms of Alzheimer’s disease (AD), yet its clinical response varies widely among individuals. This study aims to investigate the influence of CYP2D6 genetic variants on donepezil concentration, treatment response, and adverse effects in Korean patients with AD dementia. We conducted a longitudinal study involving 76 patients receiving either 5 mg or 10 mg of donepezil. Genetic testing identified 9 CYP2D6 alleles, categorizing patients by metabolizing abilities. Blood sampling for plasma concentrations of donepezil were performed at steady-state. Mini-Mental State Examination (MMSE) were conducted at 12, 24 and 36 months after the initiation of treatment. Adverse events were collected throughout the study period.Donepezil plasma concentrations differed significantly among metabolizer statuses (mean 56.8 ± 27.1 ng/ml in normal metabolizers vs. 69.6 ± 30.1 ng/ml in intermediate metabolizers, p = 0.042), but these differences did not affect cognitive function over three years as assessed by MMSE. Additionally, there was no significant correlation between donepezil plasma concentration and adverse events. Our study is the first to elucidate the associations between CYP2D6 genotype and the concentration, clinical response or adverse events of donepezil in Korean patients with AD dementia. Larger studies are necessary to fully understand the impact of CYP2D6 genetic variants on therapeutic outcomes with donepezil.

Background: Orbital medial wall fractures, which are more common than inferior wall fractures, have traditionally been difficult to diagnose with conventional radiography. As the fracture extends deep into the bony orbit, accurately visualizing internal structures becomes challenging, increasing the risk of optic nerve compression. In a previous study, the author introduced a technique for treating medial orbital wall fractures using a combined transethmoidal and transcaruncular approach. In this study, the authors hypothesized that the use of surgical navigation could enhance precision, safety, and anatomical reconstruction in this approach and employed navigation during surgery for medial orbital wall fractures and evaluated whether it improved postoperative functional and anatomical outcomes while minimizing complications. Methods: From September 2021 to November 2023, 48 patients with isolated medial wall fractures underwent surgical treatment using a combined transcaruncular approach and transethmoidal packing to repair the orbital fracture. Of these patients, 23 underwent surgery with the use of intraoperative navigation, while the other 25 did not. Intraoperative navigation was employed to facilitate precise fracture reduction and reduce the risk of optic nerve injury. The outcomes were compared by dividing the patients into two groups. Preoperative and postoperative assessments included ophthalmologic evaluations, Hertel exophthalmometry, and computed tomography-based orbital volume measurements. Results: The surgical approach with intraoperative navigation was successfully performed in all patients. Postoperative outcomes showed full recovery without residual symptoms or complications. Orbital volume measurements indicated a significant reduction in the preoperative orbital volume ratio from 109.03% to 104.80% postoperatively (p< 0.001). However, changes in the Hertel scale were not statistically significant (p = 0.086). Conclusion The integration of intraoperative navigation in medial orbital wall fracture surgery enhances the precision of medial orbital wall restoration and minimizes postoperative complications, supporting its use in the surgical management of medial orbital fractures.

Donepezil, an acetylcholinesterase inhibitor, is widely used for managing the symptoms of Alzheimer’s disease (AD), yet its clinical response varies widely among individuals. This study aims to investigate the influence of CYP2D6 genetic variants on donepezil concentration, treatment response, and adverse effects in Korean patients with AD dementia. We conducted a longitudinal study involving 76 patients receiving either 5 mg or 10 mg of donepezil. Genetic testing identified 9 CYP2D6 alleles, categorizing patients by metabolizing abilities. Blood sampling for plasma concentrations of donepezil were performed at steady-state. Mini-Mental State Examination (MMSE) were conducted at 12, 24 and 36 months after the initiation of treatment. Adverse events were collected throughout the study period.Donepezil plasma concentrations differed significantly among metabolizer statuses (mean 56.8 ± 27.1 ng/ml in normal metabolizers vs. 69.6 ± 30.1 ng/ml in intermediate metabolizers, p = 0.042), but these differences did not affect cognitive function over three years as assessed by MMSE. Additionally, there was no significant correlation between donepezil plasma concentration and adverse events. Our study is the first to elucidate the associations between CYP2D6 genotype and the concentration, clinical response or adverse events of donepezil in Korean patients with AD dementia. Larger studies are necessary to fully understand the impact of CYP2D6 genetic variants on therapeutic outcomes with donepezil.

Donepezil, an acetylcholinesterase inhibitor, is widely used for managing the symptoms of Alzheimer’s disease (AD), yet its clinical response varies widely among individuals. This study aims to investigate the influence of CYP2D6 genetic variants on donepezil concentration, treatment response, and adverse effects in Korean patients with AD dementia. We conducted a longitudinal study involving 76 patients receiving either 5 mg or 10 mg of donepezil. Genetic testing identified 9 CYP2D6 alleles, categorizing patients by metabolizing abilities. Blood sampling for plasma concentrations of donepezil were performed at steady-state. Mini-Mental State Examination (MMSE) were conducted at 12, 24 and 36 months after the initiation of treatment. Adverse events were collected throughout the study period.Donepezil plasma concentrations differed significantly among metabolizer statuses (mean 56.8 ± 27.1 ng/ml in normal metabolizers vs. 69.6 ± 30.1 ng/ml in intermediate metabolizers, p = 0.042), but these differences did not affect cognitive function over three years as assessed by MMSE. Additionally, there was no significant correlation between donepezil plasma concentration and adverse events. Our study is the first to elucidate the associations between CYP2D6 genotype and the concentration, clinical response or adverse events of donepezil in Korean patients with AD dementia. Larger studies are necessary to fully understand the impact of CYP2D6 genetic variants on therapeutic outcomes with donepezil.

Background: and Purpose This study was an open-label, dose-escalation, phase 1 clinical trial to determine the safety and dose of EN001 for patients with Duchenne muscular dystrophy (DMD). EN001, developed by ENCell, are allogeneic early-passage Wharton’s jelly-derived mesenchymal stem cells that originate at the umbilical cord, with preclinical studies demonstrating their high therapeutic efficacy for DMD. Methods: This phase 1 clinical trial explored the safety and tolerability of EN001 as a potential treatment option for patients with DMD. Six pediatric participants with DMD were divided into two subgroups of equal size: low-dose EN001 (5.0×105 cells/kg) and high-dose EN001 (2.5×106 cells/kg). All participants were monitored for 12 weeks after EN001 administration to assess its safety. Dose-limiting toxicity (DLT) was evaluated across 2 weeks post administration. Exploratory efficacy was evaluated by measuring serum creatine kinase levels, and functional evaluations—including spirometry, myometry, the North Star Ambulatory Assessment, and the 6-minute walk test—were conducted at week 12 and compared with the baseline values. Results: No participants experienced serious adverse events related to EN001 injection during the 12-week follow-up period. Mild adverse events included injection-related local erythema, edema, parosmia, and headache, but DLT was not observed. Functional evaluations at week 12 revealed no significant changes from baseline. Conclusions These results demonstrated that EN001 are safe and well tolerated for patients with DMD, and did not cause serious adverse events. The efficacy of EN001 could be confirmed through larger-scale future studies that incorporate repeated dosing and have a randomized controlled trial design.

Background: Orbital medial wall fractures, which are more common than inferior wall fractures, have traditionally been difficult to diagnose with conventional radiography. As the fracture extends deep into the bony orbit, accurately visualizing internal structures becomes challenging, increasing the risk of optic nerve compression. In a previous study, the author introduced a technique for treating medial orbital wall fractures using a combined transethmoidal and transcaruncular approach. In this study, the authors hypothesized that the use of surgical navigation could enhance precision, safety, and anatomical reconstruction in this approach and employed navigation during surgery for medial orbital wall fractures and evaluated whether it improved postoperative functional and anatomical outcomes while minimizing complications. Methods: From September 2021 to November 2023, 48 patients with isolated medial wall fractures underwent surgical treatment using a combined transcaruncular approach and transethmoidal packing to repair the orbital fracture. Of these patients, 23 underwent surgery with the use of intraoperative navigation, while the other 25 did not. Intraoperative navigation was employed to facilitate precise fracture reduction and reduce the risk of optic nerve injury. The outcomes were compared by dividing the patients into two groups. Preoperative and postoperative assessments included ophthalmologic evaluations, Hertel exophthalmometry, and computed tomography-based orbital volume measurements. Results: The surgical approach with intraoperative navigation was successfully performed in all patients. Postoperative outcomes showed full recovery without residual symptoms or complications. Orbital volume measurements indicated a significant reduction in the preoperative orbital volume ratio from 109.03% to 104.80% postoperatively (p< 0.001). However, changes in the Hertel scale were not statistically significant (p = 0.086). Conclusion The integration of intraoperative navigation in medial orbital wall fracture surgery enhances the precision of medial orbital wall restoration and minimizes postoperative complications, supporting its use in the surgical management of medial orbital fractures.

Background: and Purpose This study was an open-label, dose-escalation, phase 1 clinical trial to determine the safety and dose of EN001 for patients with Duchenne muscular dystrophy (DMD). EN001, developed by ENCell, are allogeneic early-passage Wharton’s jelly-derived mesenchymal stem cells that originate at the umbilical cord, with preclinical studies demonstrating their high therapeutic efficacy for DMD. Methods: This phase 1 clinical trial explored the safety and tolerability of EN001 as a potential treatment option for patients with DMD. Six pediatric participants with DMD were divided into two subgroups of equal size: low-dose EN001 (5.0×105 cells/kg) and high-dose EN001 (2.5×106 cells/kg). All participants were monitored for 12 weeks after EN001 administration to assess its safety. Dose-limiting toxicity (DLT) was evaluated across 2 weeks post administration. Exploratory efficacy was evaluated by measuring serum creatine kinase levels, and functional evaluations—including spirometry, myometry, the North Star Ambulatory Assessment, and the 6-minute walk test—were conducted at week 12 and compared with the baseline values. Results: No participants experienced serious adverse events related to EN001 injection during the 12-week follow-up period. Mild adverse events included injection-related local erythema, edema, parosmia, and headache, but DLT was not observed. Functional evaluations at week 12 revealed no significant changes from baseline. Conclusions These results demonstrated that EN001 are safe and well tolerated for patients with DMD, and did not cause serious adverse events. The efficacy of EN001 could be confirmed through larger-scale future studies that incorporate repeated dosing and have a randomized controlled trial design.

Donepezil, an acetylcholinesterase inhibitor, is widely used for managing the symptoms of Alzheimer’s disease (AD), yet its clinical response varies widely among individuals. This study aims to investigate the influence of CYP2D6 genetic variants on donepezil concentration, treatment response, and adverse effects in Korean patients with AD dementia. We conducted a longitudinal study involving 76 patients receiving either 5 mg or 10 mg of donepezil. Genetic testing identified 9 CYP2D6 alleles, categorizing patients by metabolizing abilities. Blood sampling for plasma concentrations of donepezil were performed at steady-state. Mini-Mental State Examination (MMSE) were conducted at 12, 24 and 36 months after the initiation of treatment. Adverse events were collected throughout the study period.Donepezil plasma concentrations differed significantly among metabolizer statuses (mean 56.8 ± 27.1 ng/ml in normal metabolizers vs. 69.6 ± 30.1 ng/ml in intermediate metabolizers, p = 0.042), but these differences did not affect cognitive function over three years as assessed by MMSE. Additionally, there was no significant correlation between donepezil plasma concentration and adverse events. Our study is the first to elucidate the associations between CYP2D6 genotype and the concentration, clinical response or adverse events of donepezil in Korean patients with AD dementia. Larger studies are necessary to fully understand the impact of CYP2D6 genetic variants on therapeutic outcomes with donepezil.

Background: Orbital medial wall fractures, which are more common than inferior wall fractures, have traditionally been difficult to diagnose with conventional radiography. As the fracture extends deep into the bony orbit, accurately visualizing internal structures becomes challenging, increasing the risk of optic nerve compression. In a previous study, the author introduced a technique for treating medial orbital wall fractures using a combined transethmoidal and transcaruncular approach. In this study, the authors hypothesized that the use of surgical navigation could enhance precision, safety, and anatomical reconstruction in this approach and employed navigation during surgery for medial orbital wall fractures and evaluated whether it improved postoperative functional and anatomical outcomes while minimizing complications. Methods: From September 2021 to November 2023, 48 patients with isolated medial wall fractures underwent surgical treatment using a combined transcaruncular approach and transethmoidal packing to repair the orbital fracture. Of these patients, 23 underwent surgery with the use of intraoperative navigation, while the other 25 did not. Intraoperative navigation was employed to facilitate precise fracture reduction and reduce the risk of optic nerve injury. The outcomes were compared by dividing the patients into two groups. Preoperative and postoperative assessments included ophthalmologic evaluations, Hertel exophthalmometry, and computed tomography-based orbital volume measurements. Results: The surgical approach with intraoperative navigation was successfully performed in all patients. Postoperative outcomes showed full recovery without residual symptoms or complications. Orbital volume measurements indicated a significant reduction in the preoperative orbital volume ratio from 109.03% to 104.80% postoperatively (p< 0.001). However, changes in the Hertel scale were not statistically significant (p = 0.086). Conclusion The integration of intraoperative navigation in medial orbital wall fracture surgery enhances the precision of medial orbital wall restoration and minimizes postoperative complications, supporting its use in the surgical management of medial orbital fractures.

Background: and Purpose This study was an open-label, dose-escalation, phase 1 clinical trial to determine the safety and dose of EN001 for patients with Duchenne muscular dystrophy (DMD). EN001, developed by ENCell, are allogeneic early-passage Wharton’s jelly-derived mesenchymal stem cells that originate at the umbilical cord, with preclinical studies demonstrating their high therapeutic efficacy for DMD. Methods: This phase 1 clinical trial explored the safety and tolerability of EN001 as a potential treatment option for patients with DMD. Six pediatric participants with DMD were divided into two subgroups of equal size: low-dose EN001 (5.0×105 cells/kg) and high-dose EN001 (2.5×106 cells/kg). All participants were monitored for 12 weeks after EN001 administration to assess its safety. Dose-limiting toxicity (DLT) was evaluated across 2 weeks post administration. Exploratory efficacy was evaluated by measuring serum creatine kinase levels, and functional evaluations—including spirometry, myometry, the North Star Ambulatory Assessment, and the 6-minute walk test—were conducted at week 12 and compared with the baseline values. Results: No participants experienced serious adverse events related to EN001 injection during the 12-week follow-up period. Mild adverse events included injection-related local erythema, edema, parosmia, and headache, but DLT was not observed. Functional evaluations at week 12 revealed no significant changes from baseline. Conclusions These results demonstrated that EN001 are safe and well tolerated for patients with DMD, and did not cause serious adverse events. The efficacy of EN001 could be confirmed through larger-scale future studies that incorporate repeated dosing and have a randomized controlled trial design.