Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome SurveysORPHANET JOURNAL OF RARE DISEASESIF3.52025-03-280OAAI
Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)MOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
Fabry Disease Podocytes Reveal Ferroptosis as a Potential Regulator of Cell PathologyKIDNEY INTERNATIONAL REPORTSIF5.72025-02-010OAAI
Implementation and Evaluation of a National Multidisciplinary Kidney Genetics Clinic Network Over 10 YearsKIDNEY INTERNATIONAL REPORTSIF5.72024-08-016OAAI
Genomic Testing in Patients with Kidney Failure of an Unknown Cause A National Australian StudyCLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGYIF7.12024-05-032OAAI
Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)MOLECULAR GENETICS AND METABOLISMIF3.52024-02-012PRE
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label studyORPHANET JOURNAL OF RARE DISEASESIF3.52023-10-2119OAAI
GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individualsAMERICAN JOURNAL OF HUMAN GENETICSIF8.12023-09-016OAAI
STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase resultsMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Clinical characteristics of female patients enrolled in the FollowME Fabry Pathfinders registryMOLECULAR GENETICS AND METABOLISMIF3.52023-02-011PRE
The spectrum of podocyte injury in later onset (LO) variants of Fabry disease (FD)MOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Long-term outcomes in patients with Fabry disease who were treated with agalsidase alfa for more than nineteen years: The Fabry Outcome SurveyMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomesJOURNAL OF MEDICAL GENETICSIF3.72022-12-2115OAAI
Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry DiseaseJOURNAL OF CLINICAL MEDICINEIF2.92022-08-178OAAI
Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registryORPHANET JOURNAL OF RARE DISEASESIF3.52022-06-2037OAAI
Reduced hip bone mineral density is associated with high levels of calciprotein particles in patients with Fabry diseaseOSTEOPOROSIS INTERNATIONALIF5.42022-05-161OAAI
Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?ORPHANET JOURNAL OF RARE DISEASESIF3.52022-02-087OAAI
Long-term multisystemic efficacy with migalastat in ERT-naive and ERT-experienced patients with amenable GLA variantsMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Lyso-Gb3 as a biomarker for renal and cardiac involvement in Fabry disease: An analysis from the Fabry Outcome Survey (FOS)MOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Baseline demographics and clinical characteristics of patients enrolled in the followME Fabry Pathfinders registryMOLECULAR GENETICS AND METABOLISMIF3.52022-02-011PRE
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