A novel frameshift ACTN2 variant causes a rare adult-onset distal myopathy with multi-minicores.
Lei ChenDian-Fu ChenHai-Lin DongGong-Lu LiuZhi-Ying WuPublished in: CNS neuroscience & therapeutics (2021)
Our results expanded the phenotypes of ACTN2-related myopathy and provided helpful information to clarify the molecular mechanisms.