The blood–brain barrier is the biggest challenge to the development of drugs that target the CNS. Most capillary beds leak plasma contents between and across endothelial cells by way of window-like ...
This targeted delivery approach is designed to address the safety and efficacy challenges associated with systemic gene therapies. By focusing the treatment directly where it is needed, intrathecal ...
Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile, with ...
Intracisterna magna (ICM) injection is used as a route to deliver intrathecal gene therapy to treat neuropathies and lysosomal storage disorders. This allows requiring smaller doses than if therapy ...
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