HIGHLIGHTS
- who: Rossella Parini from the ATS Monza e Brianza, Via Pergolesi, Monza, Italy have published the Article: Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy, in the Journal: (JOURNAL)
- what: Brain involvement in Pompe disease remains an open issue not treatable by ERT in the current formulation and represents one of the main goals to be pursued by future research studies.
- how: The data are in line with these previous findings.
SUMMARY
Patients with high titer were mainly cross . . .
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