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New data show children with higher baseline adaptative functioning scores, as well as girls, were more likely to have nonpersistent autism spectrum disorder in later years.
This follow-up study of a randomized clinical trial examines the effect of minimally invasive surfactant therapy, surfactant administration via a thin catheter,
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Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function...
Denali Therapeutics Inc. announced new interim data from the ongoing open-label, single-arm Phase 1/2 study of DNL310 in children with MPS II . DNL310 is an investigational enzyme replacement therapy...