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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.

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Title: Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
Authors: Ramos DM; Department of Neuroscience and., d'Ydewalle C; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Gabbeta V; PTC Therapeutics, South Plainfield, New Jersey, USA., Dakka A; PTC Therapeutics, South Plainfield, New Jersey, USA., Klein SK; Ionis Pharmaceuticals, Carlsbad, California, USA., Norris DA; Ionis Pharmaceuticals, Carlsbad, California, USA., Matson J; Ionis Pharmaceuticals, Carlsbad, California, USA., Taylor SJ; PharmOptima, Portage, Michigan, USA., Zaworski PG; PharmOptima, Portage, Michigan, USA., Prior TW; Center for Human Genetics, Case Western Reserve University, Cleveland, Ohio, USA., Snyder PJ; Department of Pathology, Ohio State University, Columbus, Ohio, USA., Valdivia D; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Hatem CL; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Waters I; The Sidney Kimmel Comprehensive Cancer Center, Department of Oncology, and., Gupte N; Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Swoboda KJ; Center for Genomic Medicine, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA., Rigo F; Ionis Pharmaceuticals, Carlsbad, California, USA., Bennett CF; Ionis Pharmaceuticals, Carlsbad, California, USA., Naryshkin N; PTC Therapeutics, South Plainfield, New Jersey, USA., Paushkin S; SMA Foundation, New York, New York, USA., Crawford TO; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.; Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Sumner CJ; Department of Neuroscience and.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Source: The Journal of clinical investigation [J Clin Invest] 2019 Nov 01; Vol. 129 (11), pp. 4817-4831.
Publication Type: Clinical Trial; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
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  Data: <searchLink fieldCode="AU" term="%22Ramos+DM%22">Ramos DM</searchLink>; Department of Neuroscience and.<br /><searchLink fieldCode="AU" term="%22d'Ydewalle+C%22">d'Ydewalle C</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Gabbeta+V%22">Gabbeta V</searchLink>; PTC Therapeutics, South Plainfield, New Jersey, USA.<br /><searchLink fieldCode="AU" term="%22Dakka+A%22">Dakka A</searchLink>; PTC Therapeutics, South Plainfield, New Jersey, USA.<br /><searchLink fieldCode="AU" term="%22Klein+SK%22">Klein SK</searchLink>; Ionis Pharmaceuticals, Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Norris+DA%22">Norris DA</searchLink>; Ionis Pharmaceuticals, Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Matson+J%22">Matson J</searchLink>; Ionis Pharmaceuticals, Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Taylor+SJ%22">Taylor SJ</searchLink>; PharmOptima, Portage, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Zaworski+PG%22">Zaworski PG</searchLink>; PharmOptima, Portage, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Prior+TW%22">Prior TW</searchLink>; Center for Human Genetics, Case Western Reserve University, Cleveland, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Snyder+PJ%22">Snyder PJ</searchLink>; Department of Pathology, Ohio State University, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Valdivia+D%22">Valdivia D</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Hatem+CL%22">Hatem CL</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Waters+I%22">Waters I</searchLink>; The Sidney Kimmel Comprehensive Cancer Center, Department of Oncology, and.<br /><searchLink fieldCode="AU" term="%22Gupte+N%22">Gupte N</searchLink>; Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Swoboda+KJ%22">Swoboda KJ</searchLink>; Center for Genomic Medicine, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Rigo+F%22">Rigo F</searchLink>; Ionis Pharmaceuticals, Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Bennett+CF%22">Bennett CF</searchLink>; Ionis Pharmaceuticals, Carlsbad, California, USA.<br /><searchLink fieldCode="AU" term="%22Naryshkin+N%22">Naryshkin N</searchLink>; PTC Therapeutics, South Plainfield, New Jersey, USA.<br /><searchLink fieldCode="AU" term="%22Paushkin+S%22">Paushkin S</searchLink>; SMA Foundation, New York, New York, USA.<br /><searchLink fieldCode="AU" term="%22Crawford+TO%22">Crawford TO</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.; Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Sumner+CJ%22">Sumner CJ</searchLink>; Department of Neuroscience and.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
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