Clinical and genetic interpretation of uncertain DMD missense variants: evidence from mRNA and protein studies.
Zhiying XieChang LiuHaiyan YuZhihao XieChengyue SunYing ZhuXiaoyu HuLi BaiLuhua WeiPeng SunYanyu LuYunlong LuYawen ZhaoWei ZhangZhaoxia WangLingchao MengYun YuanPublished in: Orphanet journal of rare diseases (2024)
Our study highlights the importance of muscle biopsy and aberrant splicing for clinical and genetic interpretation of uncertain DMD missense variants.