Pilot trial of phenylbutyrate in spinal muscular atrophy

Neuromuscular Disorders

Volumn 14, Issue 2, 2004, Pages 130-135

  Mercuri, Eugenio ^a,b   Bertini, Enrico ^c   Messina, Sonia ^d   Pelliccioni, Marco ^e   D'Amico, Adele ^a   Colitto, Francesca ^a   Mirabella, Massimiliano ^a   Tiziano, Francesco D ^a   Vitali, Tiziana ^a   Angelozzi, Carla ^a   Kinali, Maria ^b   Main, Marion ^b   Brahe, Christina ^a  

^a CATHOLIC UNIVERSITY   (Italy)

^b HAMMERSMITH HOSPITAL   (United Kingdom)

^c BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^d UNIVERSITY OF MESSINA   (Italy)

^e UILDM   (Italy)

Author keywords

Forced vital capacity; Phenylbutyrate; Spinal muscular atrophy

Indexed keywords

BUTYRIC ACID DERIVATIVE; PHENYLBUTYRATE; TRI BUTYRATE; UNCLASSIFIED DRUG;

ARTICLE; AUTOSOMAL RECESSIVE DISORDER; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG EFFICACY; DRUG TOLERABILITY; FORCED EXPIRATORY VOLUME; HUMAN; MOTOR PERFORMANCE; MUSCLE CONTRACTURE; MUSCLE EXERCISE; MUSCLE STRENGTH; PILOT STUDY; POWDER; PRIORITY JOURNAL; RASH; SIDE EFFECT; SPINAL MUSCULAR ATROPHY; STOMACH PAIN; SWEAT; TABLET; TASTE; TREATMENT OUTCOME;

EID: 9144269242     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2003.11.006     Document Type: Article

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