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Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease

  • Published:2011-07 Issue:7 Volume:21 Page:477-482
  • ISSN:0960-8966
  • Container-title:Neuromuscular Disorders
  • language:en
  • Short-container-title:Neuromuscular Disorders

Author:

Orlikowski David,Pellegrini Nadine,Prigent Hélène,Laforêt Pascal,Carlier Robert,Carlier Pierre,Eymard Bruno,Lofaso Frédéric,Annane Djillali

Publisher

Elsevier BV

Subject

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

Reference28 articles.

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

2. Towards a molecular therapy for glycogen storage disease type II (Pompe disease);Chen;Mol Med Today,2000

3. Late onset Pompe disease: clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients;Muller-Felber;Neuromuscul Disord,2007

4. Pompe’s disease;van der Ploeg;Lancet,2008

5. Juvenile and adult-onset acid maltase deficiency in France. genotype-phenotype correlation;Laforet;Neurology,2000

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