Antisense drugs for rare and ultra-rare genetic neurological diseases.

Neuron

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA. Electronic address:

Published: August 2023

The regulatory approvals of nusinersen and tofersen, plus the large body of clinical and preclinical data from other drugs, have significantly de-risked antisense technology for neurological diseases. The platform learnings over the last 2 decades can be applied to subsequent drugs to improve the efficiency of discovering effective neuro-therapeutics.

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http://dx.doi.org/10.1016/j.neuron.2023.05.027DOI Listing

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