Abstract：
Neuromuscular diseases are severe disorders affecting the peripheral nervous system, usually driving to death in a limited time. Many new drugs, through RNA-interference technology, are revolutionizing the prognosis and quality of life for these patients. Nevertheless, given the increased life expectancy, some new issues and phenotypes are expected to be revealed. In the transthyretin-mediated hereditary amyloidosis (ATTR-v, \"v\" for \"variant\"), the RNA interference was demonstrated to effectively reduce the hepatic synthesis of transthyretin, with a significant increase in disease progression in terms of polyneuropathy and cardiomyopathy. The increased life expectancy could promote the involvement of organs where the extra-hepatic transthyretin is deposited, such as the brain and eye, which are probably not targeted by the available treatments. All these issues are discussed in this editorial.
摘要：
神经肌肉疾病是影响周围神经系统的严重疾病，通常在有限的时间内开车致死。许多新药,通过RNA干扰技术，正在彻底改变这些患者的预后和生活质量。然而，考虑到预期寿命的增加，一些新的问题和表型有望被揭示。在转甲状腺素蛋白介导的遗传性淀粉样变性（ATTR-v，\"v\"for\"variant\"),RNA干扰被证明可以有效地减少肝内转录蛋白的合成，在多发性神经病和心肌病方面，疾病进展显着增加。预期寿命的增加可以促进肝外甲状腺素运载蛋白沉积的器官的参与，比如大脑和眼睛，可能不是可用治疗的目标。所有这些问题都在这篇社论中讨论。
