Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.

ABSTRACT:

Background

Early initiation of enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase is an effective treatment for patients with infantile-onset Pompe disease (IOPD) but cannot prevent a slow progression of myopathy. Albuterol has been shown to be helpful in adult patients with Pompe disease, and therefore, we administered an open-label adjunctive therapy with albuterol in IOPD patients undergoing ERT.

Methods

Fourteen patients, aged 2 to 12 years, were enrolled in this study; all of them had a disease onset before 12 months of life, and 13 of them were ambulatory because of early initiation of ERT. All patients received albuterol (also referred to as salbutamol) 12 mg daily for 26 weeks. The outcome measurements included a 6-minute walk test, four-s

SUBMITTER: Chien YH 

PROVIDER: S-EPMC5406275 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature