SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy
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Spinal muscular atrophy: from gene discovery to clinical trialsHistone deacetylase inhibitors as potential treatment for spinal muscular atrophySpinal muscular atrophy: development and implementation of potential treatmentsApplicability of histone deacetylase inhibition for the treatment of spinal muscular atrophyThe promise and perils of HDAC inhibitors in neurodegenerationDrosophila as an In Vivo Model for Human Neurodegenerative DiseaseValproic acid causes proteasomal degradation of DICER and influences miRNA expressionNew therapeutic approaches to spinal muscular atrophy.Therapeutic strategies for the treatment of spinal muscular atrophy.VPA response in SMA is suppressed by the fatty acid translocase CD36.SMN-inducing compounds for the treatment of spinal muscular atrophy.Spinal Muscular Atrophy Therapeutics: Where do we Stand?SMA-MAP: a plasma protein panel for spinal muscular atrophySpinal muscular atrophy: diagnosis and management in a new therapeutic eraAdvances in therapeutic development for spinal muscular atrophy.Epigenetic mechanisms underlying the pathogenesis of neurogenetic diseasesResistance strength training exercise in children with spinal muscular atrophyPopulation pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patientsTherapy development for spinal muscular atrophy in SMN independent targets.Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy ControlsContent validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.Spinal muscular atrophy: an update on therapeutic progressSMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophyA plural role for lipids in motor neuron diseases: energy, signaling and structure.Spinal Muscular Atrophy Biomarker Measurements from Blood Samples in a Clinical Trial of Valproic Acid in Ambulatory Adults.Spinal muscular atrophy and the antiapoptotic role of survival of motor neuron (SMN) protein.Developing therapies for spinal muscular atrophy.Dysfunction of autophagy as the pathological mechanism of motor neuron disease based on a patient-specific disease model.Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors.Splice-Switching Therapy for Spinal Muscular Atrophy.Long-term exercise-specific neuroprotection in spinal muscular atrophy-like mice.Old measures and new scores in spinal muscular atrophy patients.Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585.Application of urine cells in drug intervention for spinal muscular atrophy.Treatment Advances in Spinal Muscular Atrophy.Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.Treating pediatric neuromuscular disorders: The future is now.
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P2860
SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy
description
2011 nî lūn-bûn
@nan
2011 թուականին հրատարակուած գիտական յօդուած
@hyw
2011 թվականին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@ast
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en-gb
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@nl
type
label
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@ast
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en-gb
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@nl
altLabel
SMA CARNI-VAL TRIAL PART II: A ...... n with Spinal Muscular Atrophy
@en
prefLabel
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@ast
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en-gb
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@nl
P2093
P2860
P50
P1433
P1476
SMA CARNIVAL TRIAL PART II: a ...... n with spinal muscular atrophy
@en
P2093
Bakri Elsheik
Bernard LaSalle
Charles B Scott
Gary M Chan
Guy D'Anjou
Gyula Acsadi
Jo Anne Maczulski
John T Kissel
Mark B Bromberg
Mary K Schroth
P2860
P304
P356
10.1371/JOURNAL.PONE.0021296
P407
P577
2011-01-01T00:00:00Z