A 4-Year Observational Clinical Study of 24 Adult Pompe Disease Patients under Alglucosidase Alfa Enzyme Replacement Therapy

Name: Work Video:
Duration: 3 min 30 s
Description: BACKGROUNDLate-onset Pompe disease is a metabolic myopathy caused by a defi ciency of acid alpha glucosidase and is primarily characterised by progressive muscle weakness and loss of respiratory function.Alglucosidase alfa enzyme replacement therapy (ERT) is a licensed treatment for this condition.
Karolina M. Stępień; J. L. Whitby; Mark Roberts; Reena Sharma
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