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POMPE

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

2021-08-01

The goal of this study was to look at ERT-related skeletal muscle alterations as well as the connection between morphological/biochemical changes and clinical outcomes in patients with late-onset Pompe disease (LOPD). In the majority of LOPD patients, ERT improved skeletal muscle pathology, as well as motor and respiratory outcomes.

POMPE

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder

2020-09-18

Enzyme replacement therapy (ERT) is the only disease-specific treatment available for Pompe disease patients. The success and limitations of ERT revealed new insights into the pathophysiology of the disease, leading the development of the next generation therapies, which have already progressed to the clinic.

POMPE

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

2018-08-01

The goal of this study was to look at ERT-related skeletal muscle alterations as well as the connection between morphological/biochemical changes and clinical outcomes in patients with late-onset Pompe disease (LOPD). In the majority of LOPD patients, ERT improved skeletal muscle pathology, as well as motor and respiratory outcomes.

Rare Diseases

Current bibliography for experts

Rare Diseases

Current bibliography for experts

POMPE

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

POMPE

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder

POMPE

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

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