Fatal systemic disorder caused by biallelic variants in FARSA.
Soo Yeon KimSaebom KoHyunook KangMan Jin KimJangsup MoonByung Chan LimKi Joong KimMurim ChoiHee-Jung ChoiJong-Hee ChaePublished in: Orphanet journal of rare diseases (2022)
Our data demonstrated the pathogenicity of biallelic variants in FARSA and suggested the implication of hypomorphic variants in severe phenotypes.