The importance of synthetic pharmacotherapy for recessive cerebellar ataxias.
Marie BeaudinNicolas DupreMario MantoPublished in: Expert review of neurotherapeutics (2024)
Advances in genetic therapies for other neurogenetic conditions have paved the way to implement feasible approaches with potential dramatic benefits. Particularly, as we develop effective treatments for these conditions, we may need to combine therapies, consider newborn testing for pre-symptomatic treatment, and optimize non-pharmacological approaches.