Longitudinal evaluation of SMN levels as biomarker for spinal muscular atrophy: results of a phase IIb double-blind study of salbutamol

Francesco Danilo Tiziano; Rosa Lomastro; Emanuela Abiusi; Maria Barbara Pasanisi; Lorena Di Pietro; Stefania Fiori; Giovanni Baranello; Corrado Angelini; Gianni Sorarù; Alessandra Gaiani; Tiziana Mongini; Liliana Vercelli; Eugenio Mercuri; Gessica Vasco; Marika Pane; Giuseppe Vita; Gianluca Vita; Sonia Messina; Roberta Petillo; Luigia Passamano; Luisa Politano; Angela Campanella; Renato Mantegazza; Lucia Morandi

doi:10.1136/jmedgenet-2018-105482

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Therapeutics

Original article

Longitudinal evaluation of SMN levels as biomarker for spinal muscular atrophy: results of a phase IIb double-blind study of salbutamol

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Francesco Danilo Tiziano Institute of Genomic Medicine, Catholic University, Roma, Italy PubMed articles Google scholar articles
Rosa Lomastro Institute of Genomic Medicine, Catholic University, Roma, Italy PubMed articles Google scholar articles
Emanuela Abiusi Institute of Genomic Medicine, Catholic University, Roma, Italy PubMed articles Google scholar articles
Maria Barbara Pasanisi Muscle Pathology and Neuroimmunology Unit, Neurological Institute Carlo Besta, Milano, Italy PubMed articles Google scholar articles
Lorena Di Pietro Institute of Genomic Medicine, Catholic University, Roma, Italy PubMed articles Google scholar articles
Stefania Fiori Institute of Genomic Medicine, Catholic University, Roma, Italy PubMed articles Google scholar articles
Giovanni Baranello Developmental Neurology Unit, Neurological Institute Carlo Besta, Milano, Italy PubMed articles Google scholar articles
Corrado Angelini IRCCS Foundation S Camillo, Lido di Venezia, Italy PubMed articles Google scholar articles
Gianni Sorarù Department of Neurosciences, University of Padova, Padua, Italy PubMed articles Google scholar articles
Alessandra Gaiani Department of Neurosciences, University of Padova, Padua, Italy PubMed articles Google scholar articles
Tiziana Mongini Neuromuscular Diseases Unit, Rita Levi Montalcini Department of Neuroscience, University of Torino, Torino, Italy PubMed articles Google scholar articles
Liliana Vercelli Neuromuscular Diseases Unit, Rita Levi Montalcini Department of Neuroscience, University of Torino, Torino, Italy PubMed articles Google scholar articles
Eugenio Mercuri Department of Pediatric Neurology, Catholic University, Roma, Italy PubMed articles Google scholar articles
Gessica Vasco Department of Pediatric Neurology, Catholic University, Roma, Italy PubMed articles Google scholar articles
Marika Pane Department of Pediatric Neurology, Catholic University, Roma, Italy PubMed articles Google scholar articles
Giuseppe Vita Cardiomyology and Medical Genetics Section, Second University of Campania Naples, Napoli, Italy Nemo Sud Clinical Centre, Messina, Italy PubMed articles Google scholar articles
Gianluca Vita Nemo Sud Clinical Centre, Messina, Italy PubMed articles Google scholar articles
Sonia Messina Cardiomyology and Medical Genetics Section, Second University of Campania Naples, Napoli, Italy Nemo Sud Clinical Centre, Messina, Italy PubMed articles Google scholar articles
Roberta Petillo Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy PubMed articles Google scholar articles
Luigia Passamano Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy PubMed articles Google scholar articles
Luisa Politano Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy PubMed articles Google scholar articles
Angela Campanella Muscle Pathology and Neuroimmunology Unit, Neurological Institute Carlo Besta, Milano, Italy PubMed articles Google scholar articles
Renato Mantegazza Muscle Pathology and Neuroimmunology Unit, Neurological Institute Carlo Besta, Milano, Italy PubMed articles Google scholar articles
Lucia Morandi Muscle Pathology and Neuroimmunology Unit, Neurological Institute Carlo Besta, Milano, Italy PubMed articles Google scholar articles

Correspondence to Professor Francesco Danilo Tiziano, Institute of Genomic Medicine, Catholic University, Roma 00168, Italy; francescodanilo.tiziano{at}unicatt.it

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Citation

Tiziano FD, Lomastro R, Abiusi E, et al

Longitudinal evaluation of SMN levels as biomarker for spinal muscular atrophy: results of a phase IIb double-blind study of salbutamol

Journal of Medical Genetics 2019;56:293-300.

Publication history

Received May 11, 2018
Revised November 10, 2018
Accepted November 30, 2018
First published December 28, 2018.

Online issue publication

April 25, 2019

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