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.
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