Rare side effects caused by hydroxychloroquine, a medication used to prevent malaria and to treat several autoimmune diseases, may mimic some symptoms of Fabry disease, a case series suggests. Hydroxychloroquine (HCQ) toxicity was associated with heart, kidney and muscle problems, as observed in Fabry disease. “A thorough investigation should be performed…
The first patient has been dosed with the second, higher dose of FLT190, Freeline Therapeutics’ investigational gene therapy for Fabry disease that’s being studied in its Phase 1/2 MARVEL-1 clinical trial. Freeline’s decision to proceed with the higher dosing level of 1.5×1012 vector genomes per kilogram of body…
Mutations likely to cause Fabry disease, particularly those associated with late-onset disease, were found to be more common in an adult population of U.K. residents than the estimated prevalence of the disease itself, a genetic analysis showed. The findings suggest late-onset Fabry disease prevalence may be higher than…
JCR Pharmaceuticals and Sumitomo Dainippon Pharma have teamed up to market and commercialize in Japan agalsidase beta BS, a biosimilar of Fabrazyme, an already approved treatment designed to replace a critical enzyme missing in people with Fabry disease. According to a company press release, “This…
Experimental gene therapy 4D-310 continues to be generally safe and has effectively increased enzyme alpha-galactosidase (Gal A) blood levels in the first three patients enrolled in 4D Molecular Therapeutics’s clinical trial. These results were announced in the presentation “An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in…
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