Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years

Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy

(2011) Daniel Forsha et al.   GENETICS IN MEDICINE

Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II

(2010) Bruno Bembi et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Where do we stand in enzyme replacement therapy in Pompe’s disease?

(2010) A.T. van der Ploeg   NEUROMUSCULAR DISORDERS

Effect of enzyme therapy in juvenile patients with Pompe disease: A three-year open-label study

(2010) C.I. van Capelle et al.   NEUROMUSCULAR DISORDERS

A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

(2010) Ans T. van der Ploeg et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial

(2009) S. Strothotte et al.   JOURNAL OF NEUROLOGY

Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease

(2008) C.I. van Capelle et al.   NEUROMUSCULAR DISORDERS