Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4
Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4 https://pediatricsnationwide.org/wp-content/uploads/2022/09/InPractice_Brief_Multimodal-Molecular_Edit-1024x783.jpg 1024 783 Abbie Miller https://pediatricsnationwide.org/wp-content/uploads/2023/05/051023BT016-Abbie-Crop.jpg
Recently published in Nature Medicine, interim results from a Phase 1/2 trial offer promising results. Limb-girdle muscular dystrophy (LGMD) 2E/R4 is a rare, progressive neuromuscular disorder caused by mutations in the β-sarcoglycan (SGCB) gene. These mutations lead to SGCB protein deficiency, ultimately resulting in muscle loss. Progressive, debilitating weakness and wasting begin in the…