Infantile-Onset Pompe Disease: The Care Beyond the Cure.
نویسندگان
چکیده
In classic infantile-onset Pompe disease (IOPD), symptoms start early in life, and there is a fatal outcome if the disease remains untreated.1 Enzyme replacement therapy (ERT) with recombinant human GAA remains the single treatment favouring a prolonged life expectancy.2–4 To our knowledge, there have been no studies investigating the role of an early and intensive global rehabilitation programme in IPOD infants treated with ERT. We present an 18-month neurorehabilitation follow-up in a child.
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ورودعنوان ژورنال:
- Journal of neuromuscular diseases
دوره 2 s1 شماره
صفحات -
تاریخ انتشار 2015