Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy

Molecular Genetics and Metabolism

Volumn 122, Issue 1-2, 2017, Pages 126-134

  Xu, Xin ^a   Wang, Amy Q ^a   Latham, Lea L ^a,b   Celeste, Frank ^a   Ciccone, Carla ^b   Malicdan, May Christine ^b   Goldspiel, Barry ^c   Terse, Pramod ^a   Cradock, James ^a   Yang, Nora ^a   Yorke, Selwyn ^d   McKew, John C ^a   Gahl, William A ^b   Huizing, Marjan ^b   Carrillo, Nuria ^a,b  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

^d New Zealand Pharmaceuticals Limited   (New Zealand)

Author keywords

First in human study; GlcNAc kinase; GNE; N acetylneuraminic acid; Pharmacokinetics; Sialylation

Indexed keywords

2 ACETYLAMINO 2 DEOXY MANNOPYRANOSE; MANNITOL; N ACETYLNEURAMINIC ACID; PLACEBO; SIALIC ACID; HEXOSAMINE; N-ACETYLMANNOSAMINE; N-ACYLMANNOSAMINE KINASE; PHOSPHOTRANSFERASE;

ADULT; AGED; ANEMIA; ARTICLE; BACKACHE; BIOSYNTHESIS; CLINICAL ARTICLE; CONNECTIVE TISSUE DISEASE; CONTROLLED STUDY; DIARRHEA; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG HALF LIFE; DRUG SAFETY; DRUG TOLERABILITY; FATIGUE; FEMALE; GASTROINTESTINAL DISEASE; GNE MYOPATHY; HEADACHE; HEMATOLOGIC DISEASE; HEPATOBILIARY DISEASE; HOMOZYGOTE; HUMAN; HYPERGLYCEMIA; HYPOCALCEMIA; HYPOTENSION; INFECTION; INFESTATION; MALE; MUSCLE WEAKNESS; MYOPATHY; NAUSEA; NEUROLOGIC DISEASE; PATHOPHYSIOLOGY; PHARYNGITIS; PHASE 1 CLINICAL TRIAL; PLASMA CONCENTRATION-TIME CURVE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RASH; SIALYLATION; SIDE EFFECT; SINGLE DRUG DOSE; SKIN DISEASE; UPPER RESPIRATORY TRACT INFECTION; VASCULAR DISEASE; ALLELE; ANIMAL; BLOOD; CLINICAL TRIAL; DISTAL MYOPATHY; DOSE RESPONSE; DRUG EFFECT; GENETICS; METABOLISM; MIDDLE AGED; MUSCLE; MUTATION; ORAL DRUG ADMINISTRATION;

ADMINISTRATION, ORAL; ADULT; AGED; ALLELES; ANIMALS; DISTAL MYOPATHIES; DOSE-RESPONSE RELATIONSHIP, DRUG; DOUBLE-BLIND METHOD; FEMALE; HEXOSAMINES; HOMOZYGOTE; HUMANS; MALE; MIDDLE AGED; MUSCLES; MUTATION; N-ACETYLNEURAMINIC ACID; PHOSPHOTRANSFERASES (ALCOHOL GROUP ACCEPTOR);

EID: 85020843401     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2017.04.010     Document Type: Article

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