Disease modifying therapy of pediatric neuromuscular diseases / 中国小儿急救医学

Neuromuscular diseases (NMDs) are a group of hereditary, commonly childhood-onset neurological disorders affecting various components of motor unit, which are often characterized by reduced muscle strength.With the rapid development of precision medicine, great progress has been made in the pharmacological treatment of NMDs, and pediatric NMDs has also entered the era of disease modifying therapy.Antisense oligonucleotides, gene therapy, small molecule compounds, monoclonal antibodies and other disease modifying therapy drugs targeting the cause and pathogenesis of the diseases are gradually applied in clinical practice.What′s more, a variety of potential new drugs and therapeutic biological products are in continuous development.Hopefully, new breakthroughs are expected to be achieved in the field of pediatric NMDs treatment in the near future.

Development and current situation of reconstruction methods following total sacrectomy / 中国修复重建外科杂志

Objective: To review the development of the reconstruction methods following total sacrectomy, and to provide reference for finding a better reconstruction method following total sacrectomy. Methods: The case reports and biomechanical and finite element studies of reconstruction following total sacrectomy at home and abroad were searched. Development and current situation were summarized. Results: After developing for nearly 30 years, great progress has been made in the reconstruction concept and fixation techniques. The fixation methods can be summarized as the following three strategies: spinopelvic fixation (SPF), posterior pelvic ring fixation (PPRF), and anterior spinal column fixation (ASCF). SPF has undergone technical progress from intrapelvic rod and hook constructs to pedicle and iliac screw-rod systems. PPRF and ASCF could improve the stability of the reconstruction system. Conclusion: Reconstruction following total sacrectomy remains a challenge. Reconstruction combining SPF, PPRF, and ASCF is the developmental direction to achieve mechanical stability. How to gain biological fixation to improve the long-term stability is an urgent problem to be solved.