Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents | Orphanet Journal of Rare Diseases | Full Text
Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents | Orphanet Journal of Rare Diseases | Full Text
Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents | Orphanet Journal of Rare Diseases | Full Text,ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice - ScienceDirect,PSYCHIC FEVER (@psyfe_member) / X,Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents | Orphanet Journal of Rare Diseases | Full Text,ER‐phagy: selective autophagy of the endoplasmic reticulum | EMBO reports