A Troublesome Debate: When to Start Treatment in Adult Pompe Patients?
نویسنده
چکیده
Based on those guidelines, distinct scenarios will be discussed. Scenario 1: When shall we start treatment in an asymptomatic Pompe patient with hyperCKemia only? Here, most Pompe experts would recommend a wait-and-see strategy with close clinical follow-up every 3 months with functional measures, such as sixminute walking test, lung function tests, and a muscle MRI investigation at least every 6 months, and conceivably a sleep lab night. Scenario 2: In any case of sole or combined hypoventilation as a sign of diaphragmatic insuffi ciency, a clear recommendation for A Troublesome Debate: When to Start Treatment in Adult Pompe Patients?
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ورودعنوان ژورنال:
- Journal of neuromuscular diseases
دوره 2 s1 شماره
صفحات -
تاریخ انتشار 2015