Caiza-Zambrano, Francisco; Aldecoa, Mayra; Rugilo, Carlos; Taratuto, Ana Lía; Marchesoni, Cintia; León-Cejas, Luciana; Reisin, Ricardo; Bonardo, Pablo
(BMJ Pub. Group, 2025-02-16)
Adult polyglucosan body disease is a rare genetic condition caused by biallelic pathogenic variants in GBE-1 gene. Affected patients typically have urinary dysfunction, progressive gait disturbance and cognitive impairment. ...