NDLI: Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.

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Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.

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Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Zanetta, Chiara Nizzardo, Monica Simone, Chiara Monguzzi, Erika Bresolin, Nereo Comi, Giacomo P. Corti, Stefania
Description	Country affiliation: Italy Author Affiliation: Zanetta C ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Nizzardo M ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Simone C ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Monguzzi E ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Bresolin N ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Comi GP ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.); Corti S ( Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. Electronic address: stefania.corti@unimi.it.)
Abstract	BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by mutations in the survival motor neuron gene (SMN1) and the leading genetic cause of infant mortality. Currently, there is no effective treatment other than supportive care. OBJECTIVE: This article provides a general overview of the main aspects that need to be taken into account to design a more efficient clinical trial and to summarize the most promising molecular trials that are currently in development or are being planned for the treatment of SMA. METHODS: A systematic review of the literature was performed, identifying key clinical trials involving novel molecular therapies in SMA. In addition, abstracts presented at the meetings of the Families of Spinal Muscular Atrophy were searched and the Families of Spinal Muscular Atrophy Web site was carefully analyzed. Finally, a selection of SMA clinical trials registered at clinical-trials.gov has been included in the article. RESULTS: The past decade has seen a marked advancement in the understanding of both SMA genetics and molecular mechanisms. New molecules targeting SMN have shown promise in preclinical studies, and various clinical trials have started to test the drugs that were discovered through basic research. CONCLUSIONS: Both preclinical and early clinical trial results involving novel molecular therapies suggest that the clinical care paradigm in SMA will soon change.
File Format	HTM / HTML
ISSN	01492918
Issue Number	1
Volume Number	36
e-ISSN	1879114X
Journal	Clinical Therapeutics
Language	English
Publisher	Elsevier
Publisher Date	2014-01-01
Publisher Place	United States
Access Restriction	One Nation One Subscription (ONOS)
Subject Keyword	Discipline Pharmacology Genetic Therapy Muscular Atrophy, Spinal Genetics Therapy Survival Of Motor Neuron 1 Protein Clinical Trials As Topic Humans Molecular Targeted Therapy Drug Therapy Survival Of Motor Neuron 2 Protein Journal Article Research Support, Non-u.s. Gov't Review
Content Type	Text
Resource Type	Article
Subject	Pharmacology Pharmacology (medical)

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