Muscle Weakness Clinical Trial
Official title:
Safety and Effectiveness of Resistance Exercise Training in Patients With Late Onset Pompe Disease- A Pilot Study
The purpose of this research study is to determine if exercise will help improve muscle strength, endurance, and quality of life in individuals with Pompe disease. This is a research study to further define the outcome of patients with Pompe disease treated with a combined diet and exercise therapy.
Eligibility: Subjects that are age 16 years or older, have been diagnosed with Pompe disease
by genetic testing, and are showing symptoms such as muscle weakness
Time Commitment:
The study will last for 8 months. This study consists of a baseline visit of and return
visits at week 9, week 16, week 24, and week 32. At the week 9 visit the subject will be
given an individualized exercise plan which will be carried out over the next 24 weeks. The
subject will be asked to return for three more visits at week16, week 24 and week 32. The 24
week training period will involve 3 workouts a week approximately 15-30 minutes each. It will
also involve a breathing exercise that the subject can do at home twice daily for 10-15
minutes per session. The study visits will include the exercise plan, physical exam, blood
draw and questionnaires.
Anticipated Benefits:
The possible benefits include a delay in progression of muscle weakness. The knowledge gained
from this study will help researchers understand the effect of exercise on the disease. This
may eventually lead to new forms of prevention of symptom onset in the future.
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