Clinical trials in spinal muscular atrophy

Current Opinion in Pediatrics

Volumn 19, Issue 6, 2007, Pages 675-679

  Darras, Basil T ^a,b   Kang, Peter B ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

^b BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

Clinical trials; SMN gene; Spinal muscular atrophy; Survival motor neuron gene

Indexed keywords

ARYLBUTYRIC ACID DERIVATIVE; BUTYRIC ACID; GABAPENTIN; HISTONE DEACETYLASE INHIBITOR; HYDROXYUREA; PLACEBO; RILUZOLE; RNA; SURVIVAL MOTOR NEURON PROTEIN; VALPROIC ACID;

CELL ORGANELLE; CELL TYPE; CLINICAL RESEARCH; CLINICAL TRIAL; DRUG APPROVAL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; GENETIC ASSOCIATION; GENETIC TRANSCRIPTION; GENETICS; HUMAN; MOTOR PERFORMANCE; PILOT STUDY; PRIORITY JOURNAL; REVIEW; RNA SPLICING; SPINAL MUSCULAR ATROPHY; UNITED STATES;

ANIMALS; CHILD; CLINICAL TRIALS AS TOPIC; CYCLIC AMP RESPONSE ELEMENT-BINDING PROTEIN; ENZYME INHIBITORS; HUMANS; HYDROXYUREA; INFANT; MOTOR NEURONS; NERVE TISSUE PROTEINS; NUCLEIC ACID SYNTHESIS INHIBITORS; PHENYLBUTYRATES; RNA-BINDING PROTEINS; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD; VALPROIC ACID;

EID: 36349023761     PISSN: 10408703     EISSN: None     Source Type: Journal    
DOI: 10.1097/MOP.0b013e3282f1884c     Document Type: Review

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