Concepedia

Prognostic factors for the late onset Pompe disease with enzyme replacement therapy: From our experience of 4 cases including an autopsy case

Hiroshi Kobayashi, Yohta Shimada, Masahiro Ikegami, Toshinao Kawai, Ken Sakurai, Takashi Urashima, Masatoshi Ijima, Masako Fujiwara, Eiko Kaneshiro, Toya Ohashi,

Molecular Genetics and Metabolism · 2010 · 58 citations · 18 references

DOI

Concepts

References

18

α-Glucosidase deficiency in generalized glycogen-storage disease (Pompe's disease)

Hers Hg · Biochemical Journal · 1963 · 865 citations · Full text

, , +12

Recombinant human acid α-glucosidase

Priya S. Kishnani, Deya Corzo, Marc Nicolino et al. · Neurology · 2006 · 772 citations · Full text

, , +10

Clinical trial in duchenne dystrophy. I. The design of the protocol

Michael H. Brooke, Robert C. Griggs, Jerry R. Mendell et al. · Muscle & Nerve · 1981 · 563 citations

, , +8

Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients

M.L.C. Hagemans · Brain · 2005 · 366 citations · Full text

, , +16

Management of Progressive Muscular Dystrophy of Childhood

Paul J. Vignos · JAMA · 1963 · 326 citations

, , +16