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Title
New Therapeutic Approaches to Spinal Muscular Atrophy
Authors
Keywords
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Journal
Current Neurology and Neuroscience Reports
Volume 12, Issue 1, Pages 42-53
Publisher
Springer Nature
Online
2011-12-02
DOI
10.1007/s11910-011-0240-9
References
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Related references
Note: Only part of the references are listed.- Skeletal Muscle in Motor Neuron Diseases: Therapeutic Target and Delivery Route for Potential Treatments
- (2012) Luc Dupuis et al. CURRENT DRUG TARGETS
- Spinal Muscular Atrophy
- (2011) Stephen J. Kolb ARCHIVES OF NEUROLOGY
- Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid
- (2011) Illora A Darbar et al. BMC Neurology
- Large-scale population screening for spinal muscular atrophy: Clinical implications
- (2011) Shay Ben-Shachar et al. GENETICS IN MEDICINE
- Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy
- (2011) Cathleen M. Lutz et al. JOURNAL OF CLINICAL INVESTIGATION
- Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway
- (2011) Faraz Farooq et al. JOURNAL OF CLINICAL INVESTIGATION
- Fatigue leads to gait changes in spinal muscular atrophy
- (2011) Jacqueline Montes et al. MUSCLE & NERVE
- Reliability of the modified hammersmith functional motor scale in young children with spinal muscular atrophy
- (2011) Kristin J. Krosschell et al. MUSCLE & NERVE
- Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- (2011) Yimin Hua et al. NATURE
- Targeting RNA to treat neuromuscular disease
- (2011) Francesco Muntoni et al. NATURE REVIEWS DRUG DISCOVERY
- Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy
- (2011) George Z. Mentis et al. NEURON
- 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
- Stem cell technology for the study and treatment of motor neuron diseases
- (2011) J Simon Lunn et al. Regenerative Medicine
- Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
- (2011) M. A. Passini et al. Science Translational Medicine
- Outcome of goal-directed non-invasive ventilation and mechanical insufflation/exsufflation in spinal muscular atrophy type I
- (2010) M. Chatwin et al. ARCHIVES OF DISEASE IN CHILDHOOD
- Association of Plastin 3 Expression With Disease Severity in Spinal Muscular Atrophy Only in Postpubertal Females
- (2010) George Stratigopoulos et al. ARCHIVES OF NEUROLOGY
- New insights into the pathogenesis of spinal muscular atrophy
- (2010) Yasushi Ito et al. BRAIN & DEVELOPMENT
- Riluzole pharmacokinetics in young patients with spinal muscular atrophy
- (2010) Chadi Abbara et al. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
- The genetics of spinal muscular atrophies
- (2010) Claribel D Wee et al. CURRENT OPINION IN NEUROLOGY
- Noninvasive Treatment Strategy for Swallowing Problems Related to Prolonged Nonoral Feeding in Spinal Muscular Atrophy Type II
- (2010) Tae-Hyun Cha et al. DYSPHAGIA
- Stem cell-derived neurotrophic support for the neuromuscular junction in spinal muscular atrophy
- (2010) Tanya J Wyatt et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development
- (2010) Rebecca M Pruss et al. Future Medicinal Chemistry
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- Antisense oligonucleotides and spinal muscular atrophy: skipping along
- (2010) A. H. M. Burghes et al. GENES & DEVELOPMENT
- SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy
- (2010) Markus Riessland et al. HUMAN MOLECULAR GENETICS
- Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
- (2010) Elisa Dominguez et al. HUMAN MOLECULAR GENETICS
- Cardiac defects contribute to the pathology of spinal muscular atrophy models
- (2010) Monir Shababi et al. HUMAN MOLECULAR GENETICS
- Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
- (2010) Adam K. Bevan et al. HUMAN MOLECULAR GENETICS
- A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype
- (2010) Jordan T. Gladman et al. HUMAN MOLECULAR GENETICS
- A feedback loop regulates splicing of the spinal muscular atrophy-modifying gene, SMN2
- (2010) Francine M. Jodelka et al. HUMAN MOLECULAR GENETICS
- Bioelectrical Impedance Analysis Can Be a Useful Screen for Excess Adiposity in Spinal Muscular Atrophy
- (2010) Douglas M. Sproule et al. JOURNAL OF CHILD NEUROLOGY
- Muscle Volume Estimation by Magnetic Resonance Imaging in Spinal Muscular Atrophy
- (2010) Douglas M. Sproule et al. JOURNAL OF CHILD NEUROLOGY
- Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design
- (2010) F. D. Tiziano et al. JOURNAL OF MEDICAL GENETICS
- In Vivo NMDA Receptor Activation Accelerates Motor Unit Maturation, Protects Spinal Motor Neurons, and Enhances SMN2 Gene Expression in Severe Spinal Muscular Atrophy Mice
- (2010) O. Biondi et al. JOURNAL OF NEUROSCIENCE
- Insights into genotype-phenotype correlations in spinal muscular atrophy: A retrospective study of 103 patients
- (2010) Florence Petit et al. MUSCLE & NERVE
- Characterizing spinal muscular atrophy with electrical impedance myography
- (2010) Seward B. Rutkove et al. MUSCLE & NERVE
- Compound muscle action potential and motor function in children with spinal muscular atrophy
- (2010) Aga Lewelt et al. MUSCLE & NERVE
- Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
- (2010) Kevin D Foust et al. NATURE BIOTECHNOLOGY
- Hydroxyurea enhances SMN2 gene expression through nitric oxide release
- (2010) Cheng Xu et al. NEUROGENETICS
- Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy
- (2010) T.- H. Chen et al. NEUROLOGY
- Assessing spinal muscular atrophy with quantitative ultrasound
- (2010) J. S. Wu et al. NEUROLOGY
- Ultrasound evaluation of fetal movements in pregnancies at risk for severe spinal muscular atrophy
- (2010) Juan Parra et al. NEUROMUSCULAR DISORDERS
- 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
- Synaptic Defects in the Spinal and Neuromuscular Circuitry in a Mouse Model of Spinal Muscular Atrophy
- (2010) Karen K. Y. Ling et al. PLoS One
- Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
- (2010) C. F. Valori et al. Science Translational Medicine
- Aquatic Therapy for a Child with Type III Spinal Muscular Atrophy: A Case Report
- (2010) Yasser Salem et al. PHYSICAL & OCCUPATIONAL THERAPY IN PEDIATRICS
- Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
- (2009) Virginia B. Mattis et al. HUMAN MOLECULAR GENETICS
- 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
- Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy
- (2009) M. L. Hastings et al. Science Translational Medicine
- Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy
- (2008) Ferrill F. Rose et al. HUMAN MOLECULAR GENETICS
- Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice
- (2008) Li-Kai Tsai et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Plastin 3 Is a Protective Modifier of Autosomal Recessive Spinal Muscular Atrophy
- (2008) G. E. Oprea et al. SCIENCE
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