Challenges and opportunities in clinical trials for spinal muscular atrophy

Neurology

Volumn 65, Issue 9, 2005, Pages 1352-1357

  Hirtz, Deborah ^a,e   Iannaccone, S ^b   Heemskerk, J ^a   Gwinn Hardy, K ^a   Moxley III, R ^c   Rowland, L P ^d  

^a NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^b UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^c UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^d Och Spine at New York Presbyterian Hospitals   (United States)

^e NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACLARUBICIN; ARYLBUTYRIC ACID DERIVATIVE; BUTYRIC ACID; CREATINE; HISTONE DEACETYLASE INHIBITOR; HYDROXYUREA; PREDNISONE;

AUTOSOMAL RECESSIVE DISORDER; CAUSE OF DEATH; CHROMOSOME 5Q; CLINICAL TRIAL; DISABILITY; DISEASE SEVERITY; DUCHENNE MUSCULAR DYSTROPHY; GENE; GENE DELETION; GENE LOCUS; GENE MUTATION; HUMAN; INFANT DISEASE; KUGELBERG WELANDER DISEASE; MORTALITY; MUSCLE STRENGTH; MUSCLE WEAKNESS; NEUROMUSCULAR DISEASE; NONHUMAN; ONSET AGE; PRIORITY JOURNAL; PROGNOSIS; RESPIRATORY FAILURE; REVIEW; SMN1 GENE; SMN2 GENE; SPINAL MUSCULAR ATROPHY; WERDNIG HOFFMANN DISEASE;

EID: 27644537716     PISSN: 00283878     EISSN: None     Source Type: Journal    
DOI: 10.1212/01.wnl.0000183282.10946.c7     Document Type: Review

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