Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study

Journal of Inherited Metabolic Disease

Volumn 36, Issue 6, 2013, Pages 1015-1024

  Borgwardt, L ^a   Dali, C I ^a   Fogh, J ^b   Mansson J E ^c   Olsen, K J ^d   Beck, H C ^e   Nielsen, K G ^a   Nielsen, L H ^a   Olsen, S O E ^a   Riise Stensland, H M F ^f   Nilssen, O ^f   Wibrand, F ^a   Thuesen, A M ^g   Pearl, T ^a   Haugsted, U ^a   Saftig, P ^h   Blanz, J ^h   Jones, S A ⁱ   Tylki Szymanska, A ^j   Guffon Fouiloux, N ^k   Beck, M ^l   Lund, A M ^a   more..

^a Rigshospitalet   (Denmark)

^b Zymenex AS   (Denmark)

^c SAHLGRENSKA UNIVERSITY HOSPITAL   (Sweden)

^d Larix CRO   (Denmark)

^e ODENSE UNIVERSITY HOSPITAL   (Denmark)

^f UNIVERSITY OF TROMSØ   (Norway)

^g Psykolog2   (Denmark)

^h UNIVERSITY OF KIEL   (Germany)

ⁱ UNIVERSITY OF MANCHESTER   (United Kingdom)

^j CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^k EDOUARD HERRIOT HOSPITAL   (France)

^l UNIVERSITY MEDICAL CENTER MAINZ   (Germany)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA MANNOSIDASE; GLIAL FIBRILLARY ACIDIC PROTEIN; OLIGOSACCHARIDE; TAU PROTEIN;

3 MINUTES STAIR CLIMB TEST; 6 MINUTES WALK TEST; ADOLESCENT; AREA UNDER THE CURVE; ARTICLE; BRUININKS OSERETSKY TEST OF MOTOR PROFICIENCY; CEREBROSPINAL FLUID LEVEL; CHILD; CHILL; CLIMBING; CLINICAL ARTICLE; COGNITION; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG SAFETY; ENZYME REPLACEMENT; FEMALE; FEVER; FOLLOW UP; FUNCTIONAL ASSESSMENT; HEADACHE; HUMAN; INFECTION; INFUSION RELATED REACTION; LEITER R TEST; LONGITUDINAL STUDY; MALE; MANNOSIDOSIS; MAXIMUM PLASMA CONCENTRATION; MOTOR PERFORMANCE; MULTIPLE DRUG DOSE; MUSCULOSKELETAL PAIN; OPEN STUDY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROTEIN BLOOD LEVEL; PROTEIN URINE LEVEL; RANDOMIZED CONTROLLED TRIAL; RESPIRATORY TRACT DISEASE; SCHOOL CHILD; SEIZURE; TIME TO MAXIMUM PLASMA CONCENTRATION; UNCONSCIOUSNESS; URTICARIA;

ADOLESCENT; ALPHA-MANNOSIDASE; ALPHA-MANNOSIDOSIS; CHILD; COGNITION; DOSE-RESPONSE RELATIONSHIP, DRUG; ENZYME REPLACEMENT THERAPY; EXERCISE TEST; FOLLOW-UP STUDIES; HUMANS; PSYCHOMOTOR PERFORMANCE; RECOMBINANT PROTEINS; TREATMENT OUTCOME;

EID: 84888198321     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-013-9595-1     Document Type: Article

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