Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study

Journal of Neuromuscular Diseases

Volumn 3, Issue 1, 2016, Pages 49-66

  Argov, Zohar ^a   Caraco, Yoseph ^a   Lau, Heather ^b   Pestronk, Alan ^c   Shieh, Perry B ^d   Skrinar, Alison ^e   Koutsoukos, Tony ^e   Ahmed, Ruhi ^e   Martinisi, Julia ^e   Kakkis, Emil ^e  

^a HEBREW UNIVERSITY OF JERUSALEM   (Israel)

^b NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e ULTRAGENYX PHARMACEUTICAL INC   (United States)

Author keywords

distal myopathy with rimmed vacuoles; GNE Myopathy; hereditary inclusion body myopathy; myopathy therapy; Sialic acid

Indexed keywords

N ACETYLNEURAMINIC ACID; PLACEBO;

ABDOMINAL PAIN; ADULT; AGED; ANXIETY; ARTHRALGIA; ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BACKACHE; CLINICAL ARTICLE; CONTROLLED STUDY; CONTUSION; COUGHING; DEPRESSION; DIARRHEA; DISEASE SEVERITY; DISTAL MYOPATHY; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DYNAMOMETRY; DYSPEPSIA; FALLING; FATIGUE; FEMALE; FLATULENCE; FLU LIKE SYNDROME; FUNCTIONAL ASSESSMENT; HEADACHE; HEREDITARY INCLUSION BODY MYOPATHY; HUMAN; HYPESTHESIA; INFORMED CONSENT; LIMB PAIN; MALE; MUSCLE STRENGTH; MUSCLE WEAKNESS; MUSCULOSKELETAL PAIN; MYALGIA; MYOPATHY; MYOPATHY WITH RIMMED VACUOLE; NAUSEA; NONAKA DISEASE; NOSE OBSTRUCTION; OROPHARYNX PAIN; PAIN; PHASE 2 CLINICAL TRIAL; POPULATION RESEARCH; PRIORITY JOURNAL; PROCEDURAL PAIN; RANDOMIZED CONTROLLED TRIAL; RASH; RHINOPHARYNGITIS; RHINORRHEA; SIDE EFFECT; TREATMENT OUTCOME; ADOLESCENT; CLINICAL TRIAL; MIDDLE AGED; MULTICENTER STUDY; OUTCOME ASSESSMENT; YOUNG ADULT;

ADOLESCENT; ADULT; DISTAL MYOPATHIES; DOUBLE-BLIND METHOD; FEMALE; HUMANS; MALE; MIDDLE AGED; N-ACETYLNEURAMINIC ACID; OUTCOME ASSESSMENT (HEALTH CARE); YOUNG ADULT;

EID: 85030419326     PISSN: 22143599     EISSN: 22143602     Source Type: Journal    
DOI: 10.3233/JND-159900     Document Type: Article

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