Search Results - "Rehborg, Rebecca"
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Assessment of cerebral spinal fluid biomarkers and microRNA-mediated disease mechanisms in spinal muscular atrophy patient samples
Published in Human molecular genetics (04-06-2022)“…Cerebral spinal fluid (CSF) is a promising biospecimen for the detection of central nervous system biomarkers to monitor therapeutic efficacy at the cellular…”
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Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
Published in The Lancet (British edition) (12-03-2022)“…Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial…”
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Outcomes of early‐treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study
Published in Muscle & nerve (01-12-2024)“…Introduction/Aims While prompt identification and treatment of infants with spinal muscular atrophy (SMA) can ameliorate outcomes, variability persists. This…”
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Validity of respiratory scores in bronchiolitis
Published in Hospital pediatrics (01-10-2012)“…The primary objective of this study was to establish the validity and reliability of 2 respiratory scores, the Respiratory Distress Assessment Instrument…”
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