FAB-GT biopsy data: 100% reduction of kidney substrate at 12 months in first patient dosed with plato ® gene therapy platform Gb3 is a fatty substrate that accumulates in the cells of Fabry disease ...
The MODIFY trial was not designed to assess renal outcomes as primary endpoints, and no statistically significant ...
Idorsia Ltd (SIX: IDIA) announces the publication of results from the pivotal Phase 3 MODIFY study and its open-label extension (OLE) evaluating lucerastat, an oral substrate reduction therapy, in ...
(RTTNews) - Idorsia Ltd (IDIA.SW) announced the publication of pivotal Phase 3 MODIFY study results and its open-label extension (OLE) in Nature Communications. The study evaluated lucerastat, an oral ...
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...
Background: Niemann-Pick type C (NPC) disease is a rare inborn error of metabolism caused by defective intracellular cholesterol traffic. Recently, substrate ...
Consistent with the 87% reduction in the first evaluable kidney biopsy, there is complete clearance of Gb3 inclusions in second evaluable kidney biopsy in Fabry disease Phase 2 trial New Gaucher ...
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