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

OBJECTIVE: To compare the short-term effectiveness of "SkyWalker" robot-assisted total knee arthroplasty (TKA) and traditional TKA. METHODS: A clinical data of 54 patients (54 knees) with TKA who met the selection criteria between January 2022 and March 2022 was retrospectively analyzed. Among them, 27 cases underwent traditional TKA (traditional operation group) and 27 cases underwent "SkyWalker" robot-assisted TKA (robot-assisted operation group). There was no significant difference between the two groups ( P>0.05) in terms of gender, age, body mass index, osteoarthritis side, disease duration, and preoperative Knee Society Score (KSS), Western Ontario and McMaster University Osteoarthritis Index (WOMAC), visual analogue scale (VAS) score, hip-knee-ankle angle (HKA), lateral distal femoral angle (LDFA), medial proximal tibial angle (MPTA), and posterior proximal tibial angle (PPTA). The operative time, intraoperative bleeding volume, surgery-related complications, the KSS, WOMAC, and VAS scores before operation and at 6 months after operation, and Forgotten Joint Score (FJS) at 6 months after operation were recorded. X-ray films were taken to review the prosthesis position and measure HKA, LDFA, MPTA, and PPTA. The differences of the clinical and imaging indicators between before and after operation were calculated and statistically analyzed. RESULTS: The operations were completed successfully in both groups. There was no significant difference in the operative time and intraoperative bleeding volume between the two groups ( P>0.05). After operation, 1 case of incision nonunion and 1 case of heart failure occurred in the traditional operation group, while no surgery-related complications occurred in the robotic-assisted operation group. The incidences of surgical complications were 7.4% (2/27) in the traditional operation group and 0 (0/27) in the robotic-assisted operation group, with no significant difference ( P=0.491). Patients in both groups were followed up 6 months. KSS score, WOMAC score, VAS score, and ROM significantly improved in both groups at 6 months after operation when compared with preoperative ones ( P<0.05). There was no significant difference between the two groups ( P>0.05) in the differences between the pre- and post-operative values of the clinical indicators and FJS scores at 6 months after operation. X-ray films showed that the lower extremity force lines of the patients improved and the knee prostheses were in good position. Except for LDFA in the robot-assisted operation group, HKA, LDFA, MPTA, and PPTA significantly improved in both groups at 6 months after operation when compared with the preoperative ones ( P<0.05). There was no significant difference between the two groups in the differences between the pre- and post-operative values of the radiological indicators ( P>0.05). CONCLUSION The "SkyWalker" robot-assisted TKA is one of the effective methods for the treatment of knee osteoarthritis and had good short-term effectiveness. But the long-term effectiveness needs to be further studied.
Humans ; Arthroplasty, Replacement, Knee/methods* ; Knee Joint/surgery* ; Knee Prosthesis ; Osteoarthritis, Knee/surgery* ; Retrospective Studies ; Robotics ; Treatment Outcome

Objective To investigate the effect and underlying mechanism of SULT2B1 in the de-velopment of atherosclerosis(AS).Methods Twelve 8-week-old apolipoprotein E-knockout(apoE-/-)male mice were subjected and fed with a high-fat diet for 12 weeks,and then randomly divided into adeno-associated virus(AAV)-GFP and AAV-shSULT2B1 groups,with 6 animals in each group.In 4 weeks after AAV injection via tail vein,the mice were sacrificed for assessing aortic and aortic root plaque formation by oil red O staining and detecting serum levels of inflam-matory factors and blood lipids.RAW264.7 cells were transfected with adenovirus(Ad)-GFP and Ad-SULT2B1,respectively(n=3).RNA sequencing was performed to detect downstream RNA changes.Then LncRNA gga3-204 was selected for downstream study.After RAW264.7 cells were divided into si-NC group,si-SULT2B1 group,si-Lncgga3-204 group and si-SULT2B1+si-Lncg-ga3-204 group(n=3),and the IL-1β and IL-6 levels were detected in these transfected cells.Results There was no statistically difference in body weight in the mice from the AAV-GFP and AAV-shSULT2B1 groups after high-fat feeding(P＞0.05).Significantly lower serum levels of TC,TG and LDL-C,reduced aortic plaque area[(8.38±1.33)％vs(11.83±1.04)％,P=0.000],and decreased TG content within the aortic root plaque[(12.29±1.54)％vs(17.67±1.53)％,P=0.000]were observed in the AAV-shSULT2B1 group than those in the AAV-GFP group.Ser-um IL-1β and IL-6 levels in the mice of the AAV-shSULT2B1 group than those in the AAV-GFP group(P＜0.01).The AAV-shSULT2B1 group also had obviously lower serum levels of I L-1 βand IL-6 than the AAV-GFP group(P＜0.01).In the RAW264.7 cells from the si-SULT2B1 group,the mRNA levels of IL-1β and IL-6 were notably lower than those in the si-NC group(P＜0.01).LncRNA gga3-204 expression was significantly higher in the Ad-shSULT2B1 group than the Ad-GFP group(P＜0.01).While,the si-SULT2B1 group had statistically higher Lncgga3-204 level than the si-NC group(2.32±0.60 vs 1.19±0.21,P=0.036).The si-Lncgga3-204 group had significantly higher IL-1β and IL-6 mRNA levels than the si-NC group(P＜0.01).The si-SULT2B1+si-Lncgga3-204 group had significantly higher IL-1β and IL-6 mRNA levels than the si-SULT2B1 group(P＜0.05).Conclusion SULT2B1 affects the macrophage inflammatory response via Lncgga3-204,and then affects the progression of AS.