Fatemeh Moosavi Author
Subjects of specialization
Affiliation
Late-onset pompe disease, Motor functions, CPK levels, Enzyme replacement therapy
Department of Community Medicine, Faculty of Medicine, Tehran Medical Sciences Branch, Islamic Azad University, Tehran, Iran
Fatemeh Moosavi, belonging to Department of Community Medicine, Faculty of Medicine, Tehran Medical Sciences Branch, intrested in the field of Enzyme replacement therapy; Late-onset pompe disease; Motor functions; CPK levels from Islamic Azad University, Tehran, Iran
Research Article Open Access
Author(s): Khadijeh Haji Naghi Tehrani, Sahar Aslzadeh, Mehrnosh Boroomandpoor, Seyed Hamid Reza Moezzi, Fatemeh Moosavi and Mehdi Vahiddastjerdi
Background: Pompe is a congenital metabolic disorder and muscles involvement that is the most common presentation of it. The late-onset Pompe disease (LOPD) is a type of Pompe according to the age of clinical presentation. In LOPD, the muscles of the trunk and proximal lower extremity have not normal function, and the repertory failure can be happening for these patients. Enzyme replacement therapy (ERT) is a new method for treatment of these patients, but the efficacy and safety of this method are challenging for long-term so that in this pilot study efficacy and safety were evaluated for two years. Methods: In this prospective study, the patients with LOPD included this study, and they received rhGAA every two weeks for two years. In this period the motor and respiratory function of t... view moreĀ»