About the LUCE-1 Trial LUCE-1 is a Phase 1/2 multicenter, open-label, dose escalation study investigating safety, tolerability and preliminary efficacy of 3 dose levels of dual AAV8.MYO7A (AAVB-081) ...
The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.
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The firm at the JP Morgan Healthcare Conference additionally expects a regulatory decision on its gene therapy RGX-121 for Hunter syndrome next month.
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Researchers are uncovering how disrupted glutamatergic synapses drive diverse forms of intellectual disability, revealing new ...
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