Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome

Proceedings of the National Academy of Sciences of the United States of America

Volumn 111, Issue 12, 2014, Pages 4596-4601

  Khwaja, Omar S ^a,b   Ho, Eugenia ^a,c   Barnes, Katherine V ^a   O'Leary, Heather M ^a   Pereira, Luis M ^a   Finkelstein, Yaron ^a,d   Nelson III, Charles A ^a   Vogel Farley, Vanessa ^a   DeGregorio, Geneva ^a   Holm, Ingrid A ^a,e   Khatwa, Umakanth ^a   Kapur, Kush ^a   Alexander, Mark E ^a,e   Finnegan, Deirdre M ^a   Cantwell, Nicole G ^a   Walco, Alexandra C ^a   Rappaport, Leonard ^a   Gregas, Matt ^a   Fichorova, Raina N ^f   Shannon, Michael W ^a   Sur, Mriganka ^g   Kaufmann, Walter E ^a   more..

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b F HOFFMANN LA ROCHE LTD   (Switzerland)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e HARVARD MEDICAL SCHOOL   (United States)

^f BRIGHAM AND WOMEN S HOSPITAL   (United States)

^g MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DIAZEPAM; ETIRACETAM; GABAPENTIN; LAMOTRIGINE; LORAZEPAM; MELATONIN; RECOMBINANT SOMATOMEDIN C; SOMATOMEDIN C;

ANXIETY; ANXIETY DISORDER; APNEA; APNEA INDEX; AREA UNDER THE CURVE; ARTICLE; BEHAVIOR DISORDER; BREATHING; CEREBROSPINAL FLUID ANALYSIS; CHILD; CLINICAL ARTICLE; CLINICAL ASSESSMENT; DEPRESSION; DRUG ABSORPTION; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; ELECTROENCEPHALOGRAM; FEMALE; GENE; GENE MUTATION; HUMAN; HYPERVENTILATION INDEX; HYPOGLYCEMIA; MAXIMUM PLASMA CONCENTRATION; MEAN RESIDENCE TIME; MECP2 GENE; MOOD; NAUSEA AND VOMITING; NONVERBAL COMMUNICATION; PATIENT COMPLIANCE; PHASE 1 CLINICAL TRIAL; PHENOTYPE; PRESCHOOL CHILD; PRIORITY JOURNAL; QUESTIONNAIRE; RESPIRATORY DISTRESS; RESPIRATORY TRACT PARAMETERS; RETT SYNDROME; SCHOOL CHILD; SOCIAL ISOLATION; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT RESPONSE;

CHILD; CHILD, PRESCHOOL; FEMALE; HUMANS; INSULIN-LIKE GROWTH FACTOR I; INTERCELLULAR SIGNALING PEPTIDES AND PROTEINS; RECOMBINANT PROTEINS; RETT SYNDROME;

EID: 84896912072     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1311141111     Document Type: Article

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