Expert Opinion on Biological Therapy, Journal Year: 2025, Volume and Issue: unknown
Published: March 5, 2025
X-linked myotubular myopathy (XLMTM) is a life-threatening congenital disorder characterized by severe respiratory and motor impairment. This disease presents significant therapeutic challenges, with various strategies being explored to address its underlying pathology. Among these approaches, gene replacement therapy has demonstrated substantial functional improvements in clinical trials. However, safety issues emerged across different highlighting the need for further research. review provides comprehensive analysis of data gathered from natural history studies, preclinical models trials, particular focus on XLMTM. The are addressed, including their outcomes associated concerns. Despite encouraging potential XLMTM, occurrence challenges emphasizes urgent more understanding disease's complex phenotype. Enhancing accurately mimic full spectrum manifestations will be crucial optimizing reducing risks future applications.
Language: Английский