Treatment with Oral ATP decreases alternating hemiplegia of childhood with de novo ATP1A3 Mutation.

ABSTRACT:

Background

Alternating hemiplegia of childhood is an intractable neurological disorder characterized by recurrent episodes of alternating hemiplegia accompanied by other paroxysmal symptoms. Recent research has identified mutations in the ATP1A3 gene as the underlying cause. Adenosine-5'-triphosphate has a vasodilatory effect, can enhance muscle strength and physical performance, and was hypothesized to improve the symptoms of paroxysmal hemiplegia.

Methods

A 7-year-old boy with alternating hemiplegia of childhood who was positive for a de novo ATP1A3 mutation was treated with adenosine- 5'- triphosphate supplementation orally as an innovative therapy for 2 years. Outcome was evaluated through the follow-up of improvement of hemiplegic episodes and psychomotor development. S

SUBMITTER: Ju J 

PROVIDER: S-EPMC4855770 | biostudies-literature | 2016 May

REPOSITORIES: biostudies-literature