Daily treatment with the investigational oral therapy venglustat in men with Fabry disease led to significant reductions in globotriaosylceramide (Gb3 or Gl-3) — the fatty molecule that accumulates to toxic levels in the disease — and no signs of disease progression. That’s according to up to three years…
A variant in the GLA gene associated with Fabry disease was found in four women with Parkinson’s disease, but they had no Fabry symptoms, according to results from a study of 236 Parkinson’s patients. According to the research team, the significance of the mutation and the relationship between the two…
The next patient to receive FLT190, an investigational gene therapy for Fabry disease, in the ongoing Phase 1/2 MARVEL-1 trial will be given a second, higher dose of therapy, accelerating an earlier trial timeline. This person was originally intended to be included in the first, lowest dose group of…
Updated findings in an ongoing Phase 1/2 clinical trial of Sangamo Therapeutics‘ gene therapy ST-920 (isaralgagene civaparvovec) shows the treatment continues to be well tolerated and effective in adults with Fabry disease. As of the Nov. 19 data cutoff date, all five treated patients enrolled showed normalized levels of…
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