A randomized trial of 4-aminopyridine in EA2 and related familial episodic ataxias.

ABSTRACT:

Objective

The therapeutic effects of 4-aminopyridine (4AP) were investigated in a randomized, double-blind, crossover trial in 10 subjects with familial episodic ataxia with nystagmus.

Methods

After randomization, placebo or 4AP (5 mg 3 times daily) was administered for 2 3-month-long treatment periods separated by a 1-month-long washout period. The primary outcome measure was the number of ataxia attacks per month; the secondary outcome measures were the attack duration and patient-reported quality of life (Vestibular Disorders Activities of Daily Living Scale [VDADL]). Nonparametric tests and a random-effects model were used for statistical analysis.

Results

The diagnosis of episodic ataxia type 2 (EA2) was genetically confirmed in 7 subjects. Patients receiving pla

SUBMITTER: Strupp M 

PROVIDER: S-EPMC3136055 | biostudies-literature | 2011 Jul

REPOSITORIES: biostudies-literature