1. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands
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Finkel, R, Bertini, Enrico Silvio, Muntoni, F, Mercuri, Eugenio Maria, Chen, K, Kirschner, J, De Lemus, M, Hogrel, Jy, Khwaja, O, Main, M, Mazzone, E, Montes, J, Ramsey, D, Sejersen, T, Sumner, C, Swoboda, K, Tiziano, Francesco Danilo, Tseng, B, Van Der Pol, L, Villerot, C, Wirth, B, Witchen, A, Yeh, Ws, and Zittersteijn, A.
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Gerontology ,medicine.medical_specialty ,business.industry ,education ,Outcome measures ,Spinal muscular atrophy ,Outcome assessment ,Settore MED/03 - GENETICA MEDICA ,medicine.disease ,humanities ,Child health ,Clinical trial ,Neurology ,Family medicine ,Pediatrics, Perinatology and Child Health ,inglese ,medicine ,Neurology (clinical) ,Pediatric Neurology ,business ,Genetics (clinical) - Abstract
209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands Richard Finkel *, Enrico Bertini , Francesco Muntoni , Eugenio Mercuri d on behalf of the ENMC SMA Workshop Study Group 1 a Nemours Children’s Hospital, Orlando, USA b UCL Institute of Child Health, Dubowitz Neuromuscular Centre, London, UK c Bambino Gesu Children’s Research Hospital, Rome, Italy d Pediatric Neurology Unit, Catholic University, Rome, Italy Received 2 March 2015
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- 2015
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