To determine the effectiveness of enzyme replacement therapy (ERT) for adults with late-onset Pompe disease
AbstractEmerging phenotypes in long-term survivors with Pompe disease on standard enzyme replacement...
textabstractBackground: Pompe disease is a rare lysosomal storage disorder characterized by muscle w...
Background: Enzyme replacement therapy (ERT) with alglucosidase alfa improves the prospect of patien...
To determine the effectiveness of enzyme replacement therapy (ERT) for adults with late-onset Pompe ...
Late-onset Pompe disease (LOPD) is an autosomal recessive disorder caused by deficiency of the enzym...
International audienceThe efficacy of enzyme replacement therapy (ERT) in patients at an advanced st...
textabstractBackground: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme...
Pompe disease (PD) is a glycogen storage disorder caused by deficient activity of acid alpha-glucosi...
In patients with late-onset Pompe disease (LOPD), the efficacy of the enzyme replacement therapy (ER...
textabstractAbstract. Background: Enzyme replacement therapy (ERT) in adults with Pompe disease, a p...
The clinical course of late-onset Pompe disease is heterogeneous, and new clinical outcome measures ...
Background: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscul...
Background: Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy ...
BACKGROUND AND PURPOSE: Pompe disease is a rare inheritable muscle disorder for which enzyme replace...
markdownabstract__Abstract__ The Erasmus MC University Medical Center has a longstanding traditio...
AbstractEmerging phenotypes in long-term survivors with Pompe disease on standard enzyme replacement...
textabstractBackground: Pompe disease is a rare lysosomal storage disorder characterized by muscle w...
Background: Enzyme replacement therapy (ERT) with alglucosidase alfa improves the prospect of patien...
To determine the effectiveness of enzyme replacement therapy (ERT) for adults with late-onset Pompe ...
Late-onset Pompe disease (LOPD) is an autosomal recessive disorder caused by deficiency of the enzym...
International audienceThe efficacy of enzyme replacement therapy (ERT) in patients at an advanced st...
textabstractBackground: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme...
Pompe disease (PD) is a glycogen storage disorder caused by deficient activity of acid alpha-glucosi...
In patients with late-onset Pompe disease (LOPD), the efficacy of the enzyme replacement therapy (ER...
textabstractAbstract. Background: Enzyme replacement therapy (ERT) in adults with Pompe disease, a p...
The clinical course of late-onset Pompe disease is heterogeneous, and new clinical outcome measures ...
Background: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscul...
Background: Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy ...
BACKGROUND AND PURPOSE: Pompe disease is a rare inheritable muscle disorder for which enzyme replace...
markdownabstract__Abstract__ The Erasmus MC University Medical Center has a longstanding traditio...
AbstractEmerging phenotypes in long-term survivors with Pompe disease on standard enzyme replacement...
textabstractBackground: Pompe disease is a rare lysosomal storage disorder characterized by muscle w...
Background: Enzyme replacement therapy (ERT) with alglucosidase alfa improves the prospect of patien...
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