Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Summary

In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated in patients with Late Onset Pompe Disease.

Timeline

Start

2013-06

Primary completion

2016-12-16

Completion

2016-12-16

Publications

• Background Koeberl DD, Li S, Dai J, Thurberg BL, Bali D, Kishnani PS. beta2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease. Mol Genet Metab. 2012 Feb;105(2):221-7. doi: 10.1016/j.ymgme.2011.11.005. Epub 2011 Nov 11.
• Koeberl DD, Case LE, Desai A, Smith EC, Walters C, Han SO, Thurberg BL, Young SP, Bali D, Kishnani PS. Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease. Mol Genet Metab. 2020 Feb;129(2):67-72. doi: 10.1016/j.ymgme.2019.12.008. Epub 2019 Dec 10.

Drugs

Indications