Medical Xpress
University College London’s National Amyloidosis Center leads a multinational team reporting that a single infusion of an in vivo gene-editing therapy (nexiguran ziclumeran) produced rapid, deep, and durable reductions in serum transthyretin for hereditary transthyretin amyloidosis with polyneuropathy, with disease measures largely stable or improved through 24 months…
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Stabilization of neuropathy scores seen after gene editing therapy for rare nerve disease
Tami Buresh
01:20
University College London’s National Amyloidosis Center leads a multinational team reporting that a single infusion of an in vivo gene-editing therapy (nexiguran ziclumeran) produced rapid, deep, and durable reductions in serum transthyretin for hereditary transthyretin amyloidosis with polyneuropathy, with disease measures largely stable or improved through 24 months…
