In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate. [electronic resource]
- Annals of neurology Jun 2006
- 970-5 p. digital
Publication Type: Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
0364-5134
10.1002/ana.20836 doi
Cyclic AMP Response Element-Binding Protein--blood GABA Agents--therapeutic use Gene Expression--drug effects Heterozygote Humans Muscular Atrophy, Spinal--drug therapy Nerve Tissue Proteins--blood Pilot Projects RNA, Messenger--blood RNA-Binding Proteins--blood SMN Complex Proteins Survival of Motor Neuron 1 Protein Survival of Motor Neuron 2 Protein Valproic Acid--therapeutic use