Childhood spinal muscular atrophy: controversies and challenges.
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Skeletal muscle training for spinal muscular atrophy type 3Spinal muscular atrophy: from gene discovery to clinical trialsHigh expression level of Tra2-β1 is responsible for increased SMN2 exon 7 inclusion in the testis of SMA miceBioenergetic status modulates motor neuron vulnerability and pathogenesis in a zebrafish model of spinal muscular atrophy.Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.A multi-source approach to determine SMA incidence and research ready populationStem cell transplantation in neurological diseases: improving effectiveness in animal modelsSpinal Muscular Atrophy Therapeutics: Where do we Stand?Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trialA Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy ControlsRevised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool.Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials.Splice-switching antisense oligonucleotides as therapeutic drugsSMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophyCardiac pathology in spinal muscular atrophy: a systematic review.Anesthesia and spinal muscle atrophy.Spinal muscular atrophy: new findings for an old pathology.The promise of futility trials in neurological diseases.Spinal muscular atrophy--recent therapeutic advances for an old challenge.Longitudinal characterization of biomarkers for spinal muscular atrophy.Developmental milestones in type I spinal muscular atrophy.Genome Editing of Monogenic Neuromuscular Diseases: A Systematic Review.Perceived efficacy of salbutamol by persons with spinal muscular atrophy: A mixed methods study.Clinical Characteristics of Spinal Muscular Atrophy in Korea Confirmed by Genetic AnalysisSpinal muscular atrophy: therapeutic strategies.Very severe spinal muscular atrophy (Type 0)SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe.Reliability and validity of outcome measures of in-hospital and at-home visits in a randomized, double-blind, placebo-controlled trial for spinal muscular atrophy.Mapping the differences in care for 5,000 spinal muscular atrophy patients, a survey of 24 national registries in North America, Australasia and Europe.Natural history of infantile-onset spinal muscular atrophy.A new self-report quality of life questionnaire for children with neuromuscular disorders: presentation of the instrument, rationale for its development, and some preliminary results.Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.Electrocardiographic Tremor as an Important Diagnostic Aid in Spinal Muscular Atrophy.Counting the cost of spinal muscular atrophy.Solving the puzzle of spinal muscular atrophy: what are the missing pieces?Quantitative muscle ultrasound measures rapid declines over time in children with SMA type 1.Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy
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P2860
Childhood spinal muscular atrophy: controversies and challenges.
description
article científic
@ca
article scientifique
@fr
articol științific
@ro
articolo scientifico
@it
artigo científico
@gl
artigo científico
@pt
artigo científico
@pt-br
artikel ilmiah
@id
artikull shkencor
@sq
artículo científico
@es
name
Childhood spinal muscular atrophy: controversies and challenges.
@en
type
label
Childhood spinal muscular atrophy: controversies and challenges.
@en
prefLabel
Childhood spinal muscular atrophy: controversies and challenges.
@en
P1433
P1476
Childhood spinal muscular atrophy: controversies and challenges.
@en
P2093
Eugenio Mercuri
Susan T Iannaccone
P304
P356
10.1016/S1474-4422(12)70061-3
P577
2012-05-01T00:00:00Z