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

  • Published:2008-06 Issue:6 Volume:18 Page:447-452
  • ISSN:0960-8966
  • Container-title:Neuromuscular Disorders
  • language:en
  • Short-container-title:Neuromuscular Disorders

Author:

van Capelle C.I.,Winkel L.P.F.,Hagemans M.L.C.,Shapira S.K.,Arts W.F.M.,van Doorn P.A.,Hop W.C.J.,Reuser A.J.J.,van der Ploeg A.T.

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference29 articles.

1. Glycogen storage disease type II: acid α-glucosidase (acid maltase) deficiency;Hirschhorn,2001

2. Acid maltase deficiency;Engel,1994

3. The natural course of non-classic Pompe’s disease; a review of 225 published cases;Winkel;J Neurol,2005

4. Clinical manifestation and natural course of late-onset Pompe’s disease in 54 Dutch patients;Hagemans;Brain,2005

5. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003

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