Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Rady Children's Institute for Genomic Medicine, |
RCV001733828 | SCV001984816 | pathogenic | Severe X-linked myotubular myopathy | 2020-08-12 | criteria provided, single submitter | clinical testing | This variant affects the canonical splice donor site of intron 2 and is therefore predicted to interfere with splicing and result in loss of normal protein function through either protein truncation or nonsense-mediated mRNA decay (NMD). This variant has not been previously reported or functionally characterized in the literature to our knowledge. However, other variants at this donor splice site, c.63+1G>A and c.63+1G>C, have been previously reported in patients with X-linked Myotubular Myopathy (PMID: 10790201, 23346162). It is absent from the gnomAD population database and thus is presumed to be rare. Based on the available evidence, the c.63+1G>T variant is classified as Pathogenic. |