In mouse models of Fabry disease, oral treatment with Genz-112638 reduced tissue Gb3 accumulation in a dosedependent manner, with additive effects in combination with ERT. It doesn't imply that the ERT also has the mutation and combination therapy is a possibility so it make sense for Amicus to try that as well.
Never followed Transkaryotic Therapies and don't know about their clinical trials. Only follow the rest of the gang because they compete with PLX.
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