Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study - Wanner, Christoph; Kimonis, Virginia; Politei, Juan; Warnock, David G.; Üçeyler, Nurcan; Frey, Aline; Cornelisse, Peter; Hughes, Derralyn (Molecular genetics and metabolism reports, 2022)
Keywords: Fabry disease; Lucerastat; MODIFY; Research Paper; Substrate reduction therapy; CTCAE; PGIC-PS; α-GAL A; LVMI; ERT; Fabry disease; LVEF; OLE; BPI-SF; GSRS; PRO; SD; SRT; PGIS-D; BPI-SF3; NeP; Gb3; CESD-R-20; IBS; Lucerastat; CKD-EPI; HbA1c; NRS-11; UT; eGFR; FABPRO-GI; NYHA; CD; CE; GI; BSS; MODIFY; AE; b.i.d; ECG; EOS; PGIS-P; EOT; SF-36v2; PGIC-DS; GCS; IRB; UCI; PK; FGID; NPSI; FD; Substrate reduction therapy