LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

Development of the pan-DAC inhibitor panobinostat (LBH589): Successes and challenges

(2009) Peter Atadja   CANCER LETTERS

Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy

(2009) Kathryn J. Swoboda et al.   PLoS One

Pharmacokinetics and tissue distribution of Kendine 91, a novel histone deacetylase inhibitor, in mice

(2008) Dorleta Otaegui et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

SMN Deficiency Causes Tissue-Specific Perturbations in the Repertoire of snRNAs and Widespread Defects in Splicing

(2008) Zhenxi Zhang et al.   CELL

Histone Deacetylase Inhibitor Panobinostat Induces Clinical Responses with Associated Alterations in Gene Expression Profiles in Cutaneous T-Cell Lymphoma

(2008) L. Ellis et al.   CLINICAL CANCER RESEARCH

Messenger RNA regulation: to translate or to degrade

(2008) Ann-Bin Shyu et al.   EMBO JOURNAL

Histone deacetylase inhibitors: possible implications for neurodegenerative disorders

(2008) Eric Hahnen et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition

(2008) Jan Hauke et al.   HUMAN MOLECULAR GENETICS

Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy

(2008) Shingo Kariya et al.   HUMAN MOLECULAR GENETICS

TDP-43 Overexpression Enhances Exon 7 Inclusion during the Survival of Motor Neuron Pre-mRNA Splicing

(2008) Jayarama Krishnan Bose et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice

(2008) Li-Kai Tsai et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Regulation of SMN Protein Stability

(2008) B. G. Burnett et al.   MOLECULAR AND CELLULAR BIOLOGY

Ubiquitin, the proteasome and protein degradation in neuronal function and dysfunction

(2008) Hwan-Ching Tai et al.   NATURE REVIEWS NEUROSCIENCE

Plastin 3 Is a Protective Modifier of Autosomal Recessive Spinal Muscular Atrophy

(2008) G. E. Oprea et al.   SCIENCE

5-(N-ethyl-N-isopropyl)-amiloride enhancesSMN2exon 7 inclusion and protein expression in spinal muscular atrophy cells

(2007) Chung-Yee Yuo et al.   ANNALS OF NEUROLOGY