Emerging therapies for childhood-onset movement disorders
Lindsey Vogt,
Vicente Quiroz,
Darius Ebrahimi-Fakhari
Abstract:Purpose of review
We highlight novel and emerging therapies in the treatment of childhood-onset movement disorders. We structured this review by therapeutic entity (small molecule drugs, RNA-targeted therapeutics, gene replacement therapy, and neuromodulation), recognizing that there are two main approaches to treatment: symptomatic (based on phenomenology) and molecular mechanism-based therapy or ‘precision medicine’ (which is disease-modifying).
Recent findings
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