An experimental genome-editing therapy safely and effectively increased levels of alpha-galactosidase A (Gal A) — which is lacking in people with Fabry disease — in a mouse model of the disease, a study has shown. By only changing the genome of about 10% of cells in the liver —…
Fabrazyme (agalsidase beta) demonstrated an acceptable safety profile and resulted in sustained reductions in globotriaosylceramide (GL-3) levels over seven years among patients with Fabry disease, a large real-world study from in Japan found. The study, “…
A Phase 3 trial evaluating lucerastat, an investigational substrate reduction therapy for Fabry disease, is fully enrolled and underway in 118 adult patients. Results from the MODIFY trial (NCT03425539) are expected toward the end of this year. Those who complete the trial…
The first patient dosed with AVR-RD-01, an investigational gene therapy being tested in the ongoing Phase 2 FAB-GT clinical trial in people with Fabry disease, showed a 100% reduction, or complete clearance of toxic substrate, in a kidney biopsy…
Fabry disease patients treated with Galafold (migalastat) had stable kidney (renal) function for up to eight and a half years — regardless of treatment status, sex, or disease characteristics — an analysis of clinical trials found. Notably, the treatment — designed only for people with certain mutations in…
A genetic screening study found a higher-than-expected prevalence of undiagnosed Fabry disease in people who had kidney failure and transplant without a known cause. When the screening was extended to related family members, additional unidentified cases were found and treated early. The findings led…
Protalix BioTherapeutics and Chiesi Global Rare Diseases have launched an expanded access program (EAP) in the U.S. for PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy…
The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and effectiveness of Galafold (migalastat)Â in people with Fabry disease with specific mutations. Importantly, the therapy’s efficacy and safety also were observed…
A single administration of the experimental gene therapy ST-920 in a Fabry disease mouse model safely and effectively increased the levels of the alpha-GalA enzyme — which is lacking in people with the genetic disorder — in…
The gene therapy candidate FLT190 — designed to increase blood levels of the enzyme alpha-GAL A, which is affected in Fabry disease — has been granted orphan drug designation by the European Commission,…
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