| Q34184793 | A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype |
| Q34490238 | A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes |
| Q39350652 | A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice |
| Q34168139 | Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy |
| Q26864605 | Applicability of histone deacetylase inhibition for the treatment of spinal muscular atrophy |
| Q40414569 | Carboxylic acid derivatives of histone deacetylase inhibitors induce full length SMN2 transcripts: a promising target for spinal muscular atrophy therapeutics. |
| Q97570201 | Current and emerging treatment options for spinal muscular atrophy |
| Q38346151 | Deficiency of the splicing factor Sfrs10 results in early embryonic lethality in mice and has no impact on full-length SMN/Smn splicing |
| Q38546908 | Developing therapies for spinal muscular atrophy |
| Q33670661 | Development of histone deacetylase inhibitors as therapeutics for neurological disease |
| Q38014955 | Developments in the discovery of drugs for spinal muscular atrophy: successful beginnings and future prospects |
| Q35216315 | Discovery, synthesis, and biological evaluation of novel SMN protein modulators |
| Q34172502 | Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development |
| Q89477851 | Drug treatment for spinal muscular atrophy types II and III |
| Q34760255 | Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds |
| Q38219414 | Epigenetic-based therapies for Friedreich ataxia |
| Q36663928 | Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy |
| Q42803814 | FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts |
| Q34805096 | Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts |
| Q26828876 | Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy |
| Q34123129 | Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay. |
| Q35175035 | Increasing expression and decreasing degradation of SMN ameliorate the spinal muscular atrophy phenotype in mice |
| Q89685622 | Integrated assessment of viral transcription, antigen presentation, and CD8+ T cell function reveal multiple limitations of class I selective HDACi during HIV-1 latency reversal |
| Q38350489 | Mechanisms and clinical significance of histone deacetylase inhibitors: epigenetic glioblastoma therapy |
| Q26752798 | Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy |
| Q38006384 | Mouse models of SMA: tools for disease characterization and therapeutic development |
| Q38197609 | Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond |
| Q34237525 | New therapeutic approaches to spinal muscular atrophy |
| Q28743986 | Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model |
| Q30743508 | Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy. |
| Q36761839 | Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy |
| Q28266165 | Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response |
| Q28271235 | SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy |
| Q34312156 | SMN-inducing compounds for the treatment of spinal muscular atrophy |
| Q40834224 | Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585. |
| Q30540653 | Sodium vanadate combined with L-ascorbic acid delays disease progression, enhances motor performance, and ameliorates muscle atrophy and weakness in mice with spinal muscular atrophy. |
| Q34460251 | Spinal Muscular Atrophy Therapeutics: Where do we Stand? |
| Q21202863 | Spinal muscular atrophy |
| Q39773596 | Spinal muscular atrophy: an update |
| Q26824894 | Spinal muscular atrophy: from gene discovery to clinical trials |
| Q35340133 | Spinal muscular atrophy: journeying from bench to bedside |
| Q33871311 | Spinal muscular atrophy: mechanisms and therapeutic strategies |
| Q34065993 | Splicing of the Survival Motor Neuron genes and implications for treatment of SMA |
| Q37800936 | Stem cell-derived neurotrophic support for the neuromuscular junction in spinal muscular atrophy. |
| Q38233117 | Targeting of histone deacetylases in brain tumors. |
| Q38104379 | The methylation hypothesis of pharmacoresistance in epilepsy |
| Q37898154 | The methylation hypothesis: do epigenetic chromatin modifications play a role in epileptogenesis? |
| Q39340546 | Therapeutic approaches for spinal muscular atrophy (SMA). |
| Q34296140 | Therapeutic strategies for the treatment of spinal muscular atrophy. |
| Q36014761 | Therapy development for spinal muscular atrophy in SMN independent targets. |
| Q34122981 | Therapy development in spinal muscular atrophy |
| Q35304142 | Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability. |
| Q28740896 | Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analyses |
| Q34306857 | VPA response in SMA is suppressed by the fatty acid translocase CD36. |