Oral cysteamine bitartrate and N-acetylcysteine for patients with infantile neuronal ceroid lipofuscinosis: A pilot study

The Lancet Neurology

Volumn 13, Issue 8, 2014, Pages 777-787

  Levin, Sondra W ^a,e   Baker, Eva H ^b   Zein, Wadih M ^c   Zhang, Zhongjian ^a   Quezado, Zenaide M N ^b,f   Miao, Ning ^b   Gropman, Andrea ^d,f   Griffin, Kurt J ^a,g   Bianconi, Simona ^a   Chandra, Goutam ^a   Khan, Omar I ^d   Caruso, Rafael C ^c,h   Liu, Aiyi ^a   Mukherjee, Anil B ^a  

^a EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c NATIONAL EYE INSTITUTE   (United States)

^d NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^e WALTER REED ARMY MEDICAL CENTER   (United States)

^f CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^g UNIVERSITY OF SOUTH DAKOTA   (United States)

^h PRINCETON UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLCYSTEINE; MERCAPTAMINE; N ACETYLASPARTIC ACID;

ARTICLE; BRAIN ATROPHY; BRAIN SIZE; BRAIN STEM; CAUDATE NUCLEUS; CEREBELLUM; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; ELECTROENCEPHALOGRAM; ELECTRON MICROSCOPY; ELECTRORETINOGRAPHY; FEMALE; FOLLOW UP; GASTROINTESTINAL SYMPTOM; GLOBUS PALLIDUS; GRAY MATTER; HUMAN; HUMAN CELL; HUMAN TISSUE; INFANT; INFANT DISEASE; INFANTILE NEURONAL CEROID LIPOFUSCINOSIS; LEUKOCYTE; MALE; MOTOR DYSFUNCTION; NEURONAL CEROID LIPOFUSCINOSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; OUTCOME ASSESSMENT; PILOT STUDY; PRIORITY JOURNAL; PUTAMEN; SIDE EFFECT; THALAMUS; WEIGHT REDUCTION; WHITE MATTER;

ACETYLCYSTEINE; ADMINISTRATION, ORAL; CHILD, PRESCHOOL; CYSTEAMINE; DRUG THERAPY, COMBINATION; ELECTROENCEPHALOGRAPHY; FEMALE; FOLLOW-UP STUDIES; HUMANS; INFANT; MALE; NEURONAL CEROID-LIPOFUSCINOSES; PILOT PROJECTS;

EID: 84904299248     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(14)70142-5     Document Type: Article

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