Neuromuscular Disorders

1 Background and Aims Charcot–Marie-Tooth (CMT) and related disorders are a group of clinically and genetically heterogeneous inherited peripheral neuropathies characterized by distal muscle weakness and atrophy, sensory impairment, areflexia, and skeletal deformities. These disorders display different patterns of inheritance, including autosomal dominant (AD), autosomal recessive (AR), and X-linked patterns. To date, more than 130 genes have been implicated in CMT [1].

TREAT-NMD is internationally recognised for its central role in addressing key challenges in the development of therapies for neuromuscular diseases. Through connecting the global neuromuscular community, sharing essential knowledge and resources, and supporting innovation, the network has contributed significantly to progress in diagnosis, care and treatment development across a range of inherited neuromuscular conditions.

Abstract Idiopathic inflammatory myopathies (IIM) are a group of rare autoimmune disorders mainly involving skeletal muscles. Telitacicept is a BAFF/APRIL inhibitor, which has a promising application in B-cell-mediated autoimmune diseases; however, its efficacy in IIM patients is still unknown. Retrospective analysis of 15 IIM patients treated with telitacicept for more than 6 months in Xiangya Hospital between November 2023 and May 2025.