Enzymatic replacement therapy in patients with late-onset Pompe disease – 6-Year follow up

ConclusionThe improvements of exercise tolerance and FVC were observed in all patients in the first three years of treatment and were the most pronounced in the longest-treated patients and with the least severe neurological and respiratory symptoms. Our research suggests that early start of the ERT results in higher improvement of respiratory and ambulation functions.
Source: Polish Journal of Neurology and Neurosurgery - Category: Neurosurgery Source Type: research