Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Medical Affairs, |
RCV001003315 | SCV001161399 | uncertain significance | Farber lipogranulomatosis | 2019-06-19 | criteria provided, single submitter | literature only | Variant c.626G>A described in Ehlert et al., 2017, DOI: 10.1002/jimd.12043 has been classified as uncertain significance. Patient is compound heterozygous for variants, c.174_175insC and c.626G>A, in the ASAH1 gene. Patient received a HSCT as the only present treatment option for Farber disease at 1.3 years. The patient died shortly after transplant due to acute respiratory distress. Although this variant is classified as uncertain significance, patients identified with this variant should be examined carely for Farber disease signs and symptoms. |