Tuesday, April 30, 2024

Emerging therapies for childhood-onset movement disorders

Vogt, Lindseya,∗; Quiroz, Vicenteb,∗; Ebrahimi-Fakhari, Dariusb,c. Emerging therapies for childhood-onset movement disorders. Current Opinion in Pediatrics 36(3):p 331-341, June 2024. | DOI: 10.1097/MOP.0000000000001354 

 We highlight reports of new small molecule drugs for Tourette syndrome, Friedreich's ataxia and Rett syndrome. We also discuss developments in gene therapy for aromatic l-amino acid decarboxylase deficiency and hereditary spastic paraplegia, as well as current work exploring optimization of deep brain stimulation and lesioning with focused ultrasound.