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

Conclusion The 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