Repurposed Deferoxamine Found to Counteract Effects of LOPD Gene Variant

Repurposed Deferoxamine Found to Counteract Effects of LOPD Gene Variant

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Repurposed Deferoxamine Found to Counteract Effects of LOPD Gene Variant

Deferoxamine, a medication normally used to remove excess iron and aluminum from the body, can counteract the effects of a disease-causing genetic variant often found in people with late-onset Pompe disease (LOPD). These findings highlight the potential of drug repurposing for the development of new therapies for LOPD, scientists said. The study, titled “Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts” and conducted by a team…

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