Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy

Carboxylic acid derivatives of histone deacetylase inhibitors induce full length SMN2 transcripts: a promising target for spinal muscular atrophy therapeutics

(2011) Didem Dayangac-Erden et al.   Archives of Medical Science

Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines

(2011) Indra Sari Kusuma Harahap et al.   BRAIN & DEVELOPMENT

Sirtuins at a glance

(2011) T. Nakagawa et al.   JOURNAL OF CELL SCIENCE

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

(2011) John T. Kissel et al.   PLoS One

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

(2010) Markus Riessland et al.   HUMAN MOLECULAR GENETICS

Quantification of SMN protein in leucocytes from spinal muscular atrophy patients: effects of treatment with valproic acid

(2010) S. Piepers et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy

(2010) Kathryn J. Swoboda et al.   PLoS One

Histone Deacetylase Inhibition Activity and Molecular Docking of (E )-Resveratrol: Its Therapeutic Potential in Spinal Muscular Atrophy

(2009) Didem Dayangaç-Erden et al.   Chemical Biology & Drug Design

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

(2009) Lutz Garbes et al.   HUMAN MOLECULAR GENETICS

Is There a Future for Histone Deacetylase Inhibitors in the Pharmacotherapy of Psychiatric Disorders?

(2009) D. R. Grayson et al.   MOLECULAR PHARMACOLOGY

Valproic acid blocks excitability in SMA type I mouse motor neurons

(2009) Kristen Rak et al.   NEUROBIOLOGY OF DISEASE

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

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

Pathogenesis of proximal autosomal recessive spinal muscular atrophy

(2008) Goran Simic   ACTA NEUROPATHOLOGICA

A preliminary report on spinal muscular atrophy lymphoblastoid cell lines: Are they an appropriate tool for drug screening?

(2008) Didem Dayangaç-Erden et al.   ADVANCES IN THERAPY

Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition

(2008) Heather L. Narver et al.   ANNALS OF NEUROLOGY

Nonsense-mediated messenger RNA decay of survival motor neuron 1 causes spinal muscular atrophy

(2008) Lars Brichta et al.   HUMAN GENETICS

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