EPI-743 reverses the progression of the pediatric mitochondrial disease-Genetically defined Leigh Syndrome

Molecular Genetics and Metabolism

Volumn 107, Issue 3, 2012, Pages 383-388

  Martinelli, Diego ^a   Catteruccia, Michela ^b   Piemonte, Fiorella ^b   Pastore, Anna ^a   Tozzi, Giulia ^b   Dionisi Vici, Carlo ^a   Pontrelli, Giuseppe ^c   Corsetti, Tiziana ^d   Livadiotti, Susanna ^c   Kheifets, Viktoria ^e   Hinman, Andrew ^e   Shrader, William D ^e   Thoolen, Martin ^e   Klein, Matthew B ^e   Bertini, Enrico ^b   Miller, Guy ^e,f  

^a BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^b Unit of Neuromuscular and Neurodegenerative Disorders   (Italy)

^c Clinical Trial Center   (Italy)

^d Central Pharmacy   (Italy)

^e BioElectron Technology Corporation   (United States)

^f STANFORD UNIVERSITY   (United States)

Author keywords

EPI 743; Leigh syndrome; Mitochondrial disease

Indexed keywords

ALPHA TOCOTRIENOL QUINONE;

ABSENCE OF SIDE EFFECTS; ADOLESCENT; AREA UNDER THE CURVE; ARTICLE; CHILD; CLINICAL ARTICLE; DISEASE COURSE; DISEASE SEVERITY; DRUG ABSORPTION; DRUG BLOOD LEVEL; DRUG DISTRIBUTION; DRUG ELIMINATION; DRUG HALF LIFE; DRUG WITHDRAWAL; FEMALE; GROSS MOTOR FUNCTION MEASURE; HUMAN; INFANT; LEIGH DISEASE; MALE; MAXIMUM PLASMA CONCENTRATION; MUSCLE HYPOTONIA; OPEN STUDY; PHASE 2 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; QUALITY OF LIFE; RATING SCALE; SCHOOL CHILD; SOMNOLENCE; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT DURATION;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; COMPASSIONATE USE TRIALS; DISEASE PROGRESSION; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; INFANT; LEIGH DISEASE; MALE; MITOCHONDRIA; PSYCHOMOTOR PERFORMANCE; QUALITY OF LIFE; SEVERITY OF ILLNESS INDEX; UBIQUINONE;

EID: 84867897915     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2012.09.007     Document Type: Article

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