A non-classical presentation of APECED in a family with heterozygous R203X AIRE gene mutation.
Giorgio RadettiA PuelR FranceschiS LonghiN GalloC BetterlePublished in: Journal of endocrinological investigation (2022)
We describe for a first time a family with heterozygous R203X AIRE mutation causing an APECED-like condition, as confirmed by presence of IFN-Abs. The unusual mild phenotype should be reassuring for the patients and assist in their clinical management.