Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.
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Neurogene reports initial data from Rett syndrome gene therapy trialNeurogene has shared initial safety and tolerability results from its ongoing Phase I/II clinical trial of NGN-401, a gene ...
Clare can't speak, write or sign due to Rett syndrome so they have been communicating ... brain development and results in severe mental and physical disability. It mainly affects girls and ...
ProQR Therapeutics has announced an $8.1 million funding boost from the Rett Syndrome Research Trust, raising the total investment to $9.1 million to advance their RNA editing collaboration.
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