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

Neuromuscular Disorders

Volumn 21, Issue 7, 2011, Pages 477-482

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

^a RAYMOND POINCARÉ HOSPITAL   (France)

^b ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^c Teaching Hospital   (France)

Author keywords

Alglucosidase alfa; Enzyme replacement therapy; Pompe disease; RhGAA

Indexed keywords

RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

ADULT; ANTIBODY PRODUCTION; ARTICLE; ARTIFICIAL VENTILATION; CLINICAL ARTICLE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG EFFECT; DRUG EFFICACY; DRUG FEVER; DRUG HYPERSENSITIVITY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENZYME REPLACEMENT; EXPLORATORY RESEARCH; FEMALE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; IMMUNE COMPLEX DISEASE; INJECTION SITE ERYTHEMA; MALE; MOTOR PERFORMANCE; MUSCLE CRAMP; MUSCLE FUNCTION; OPEN STUDY; PATIENT TRANSPORT; PRIORITY JOURNAL; PROSPECTIVE STUDY; RESPIRATORY FAILURE; RESPIRATORY FUNCTION; SIDE EFFECT; SITTING; SPIROMETRY; STANDING; SUPINE POSITION; TRACHEOSTOMY; VITAL CAPACITY;

ADULT; ALPHA-GLUCOSIDASES; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; MALE; MIDDLE AGED; MUSCLE STRENGTH; PROSPECTIVE STUDIES; RECOMBINANT PROTEINS; RESPIRATORY FUNCTION TESTS; RESPIRATORY INSUFFICIENCY; TREATMENT OUTCOME;

EID: 79960093688     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2011.04.001     Document Type: Article

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