SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy
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Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acidSMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophyDrug treatment for spinal muscular atrophy types II and IIIDrug treatment for spinal muscular atrophy types II and IIISpinal 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 DiseaseEvaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical studyEstablished Stem Cell Model of Spinal Muscular Atrophy Is Applicable in the Evaluation of the Efficacy of Thyrotropin-Releasing Hormone Analog.New 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.Spinal Muscular Atrophy Therapeutics: Where do we Stand?Vitamin D intake is inadequate in spinal muscular atrophy type I cohort: correlations with bone healthSMA-MAP: a plasma protein panel for spinal muscular atrophySpinal muscular atrophy: diagnosis and management in a new therapeutic eraReliability of the Modified Hammersmith Functional Motor Scale in young children with spinal muscular atrophy.Advances in therapeutic development for spinal muscular atrophy.The Genetics of Spinal Muscular Atrophy: Progress and Challenges.Population 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.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.Prospective cohort study of spinal muscular atrophy types 2 and 3.Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials.Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with 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.Biomarkers in rare disorders: the experience with spinal muscular atrophy.Spinal muscular atrophy and the antiapoptotic role of survival of motor neuron (SMN) protein.Anesthesia and spinal muscle atrophy.Developing therapies for spinal muscular atrophy.Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors.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.
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P2860
SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy
description
2010 nî lūn-bûn
@nan
2010 թուականին հրատարակուած գիտական յօդուած
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2010 թվականին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@ast
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en-gb
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@nl
type
label
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@ast
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en-gb
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@nl
altLabel
SMA CARNI-VAL Trial Part I: Do ...... cid in Spinal Muscular Atrophy
@en
prefLabel
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@ast
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@en-gb
SMA CARNI-VAL trial part I: do ...... cid in spinal muscular atrophy
@nl
P2093
P2860
P50
P1433
P1476
SMA CARNI-VAL trial part I: do ...... cid in 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.0012140
P407
P577
2010-01-01T00:00:00Z