Effects of exercise therapy in patients with Pompe's disease

Pompe's disease is a rare myopathy consisting of acid alpha-glucosidase enzyme deficiency, which leads to accumulation of glycogen in especially skeletal muscle cells, leading to progressive muscle weakness of mostly shoulder, hip and trunk muscles. Patients are supported with enzyme replacement therapy with beneficial effects. However these beneficial effects fade over time and patients still experience limitations due to their myopathy. The search for complementing non-medical therapies are therefore of great importance for these patients. In 2011 mildly affected patients followed a training program over 12 weeks. On the short term, training improved their endurance, muscle strength and function, core stability, fatigue and pain. With our current study we aim to evaluate the long term effects of the mentioned exercise therapy. We aim to determine whether patients continued the training program and reasons of quitting the therapy. We will also select a group of controls which didn't participate in the training program. A comparison will be made between these two groups on endurance, muscle strength and function, core stability and quality of life to evaluate whether trained patients perform better.

Continuing exercise therapy will prove beneficial effects, in Pompe patients in the long-term, on one or more measured outcomes: endurance, muscle strength, muscle function, core stability and quality of life.

Completion of measurements: within 6 months. A small delay exists due to the current corona crisis. We aim to start in september.

N/A