Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C

Journal of Inherited Metabolic Disease

Volumn 36, Issue 1, 2013, Pages 129-137

  Chien, Y H ^a   Peng, S F ^a   Yang, C C ^a   Lee, N C ^a   Tsai, L K ^a   Huang, A C ^a   Su, S C ^a   Tseng, C C ^a   Hwu, W L ^a  

^a NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

Author keywords

[No Author keywords available]

Indexed keywords

MIGLUSTAT;

ADOLESCENT; ARTICLE; CHILD; CLINICAL ARTICLE; COGNITION; COGNITIVE DEFECT; DIARRHEA; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DYSPHAGIA; GASTROINTESTINAL SYMPTOM; HUMAN; MEDICAL PARAMETERS; MENTAL HEALTH; MOTOR DYSFUNCTION; NEUROLOGIC DISEASE; NIEMANN PICK DISEASE; ONSET AGE; PARESTHESIA; PRESCHOOL CHILD; PSYCHOLOGIC TEST; SCHOOL CHILD; SCORING SYSTEM; SWALLOWING; TREATMENT DURATION; TREATMENT RESPONSE;

1-DEOXYNOJIRIMYCIN; CHILD; CHILD, PRESCHOOL; COGNITION; DEGLUTITION; DISEASE PROGRESSION; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; MALE; NERVOUS SYSTEM DISEASES; NIEMANN-PICK DISEASE, TYPE C; TIME; TREATMENT OUTCOME;

EID: 84872611654     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-012-9479-9     Document Type: Article

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