Advancements in the Management of Genetic Neuromuscular Diseases in Libya: A National Perspective
DOI:
https://doi.org/10.61856/p0nb5q03Keywords:
Neuromuscular diseases genetic Duchenne muscular dystrophy Spinal muscular dystrophy Gene therapy Exon-skipping therapy LibyaAbstract
Genetic neuromuscular diseases (NMD) are rare , disabling disorders increasingly recognized worldwide with advances in molecular diagnostics and gene-targeted therapies . In libya ,however, their recognition remains limited due to restricted access to specialized services , the aim of this study to description of libya's efforts to implement national diagnostic and management programs for neruomuscular diseases . methods of this study by review of patients screened, genetic testing results , treatments provided ( SMA, DMD), and international collaborations. , the results : over 2000 patients evaluated : SMA prevalence 3,02/100.000, DMD prevalence 7,06/100.000 males. more than 100 patients treated with Risdiplam locally ; 18 childeren received zolgensma abroad ; 36 patients start exon-skipping therapies. libya has taken steps to establish a national framework for managing NMDS, however , long -term sustainability and availability of genetic therapies remain challenges .
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