Concepedia

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

S. Strothotte, N. Strigl‐Pill, Birgit Grunert, Cornelia Kornblum, K. Eger, Carsten Wessig, Marcus Deschauer, Frank Breunig, Franz X. Glocker, Stefan Vielhaber,

Journal of Neurology · 2009 · 254 citations · 11 references

DOI

Concepts

References

11

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

Priya S. Kishnani, Wuh‐Liang Hwu, Hanna Mandel et al. · The Journal of Pediatrics · 2006 · 585 citations

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Late onset Pompe disease: Clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients

Wolfgang Müller‐Felber, Rita Horváth, Klaus Gempel et al. · Neuromuscular Disorders · 2007 · 239 citations

, , +4

Enzyme replacement therapy in late‐onset Pompe's disease: A three‐year follow‐up

Léon P. F. Winkel, Johanna M. P. van den Hout, Joep H. J. Kamphoven et al. · Annals of Neurology · 2004 · 231 citations

, , +12

Structural and functional changes of lysosomal acid alpha-glucosidase during intracellular transport and maturation.

H.A. Wisselaar, Marian A. Kroos, M M Hermans et al. · Journal of Biological Chemistry · 1993 · 180 citations · Full text

, , +17

Clinical features of late‐onset Pompe disease: A prospective cohort study

John H. J. Wokke, Diana M. Escolar, Alan Pestronk et al. · Muscle & Nerve · 2008 · 172 citations

, , +16