Literature DB >> 26045156

209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands.

Richard Finkel1, Enrico Bertini2, Francesco Muntoni3, Eugenio Mercuri4.   

Abstract

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Year:  2015        PMID： 26045156     DOI： 10.1016/j.nmd.2015.04.009

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  51 in total

1.  Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden.

Authors:  Santiago Zuluaga-Sanchez; Megan Teynor; Christopher Knight; Robin Thompson; Thomas Lundqvist; Mats Ekelund; Annabelle Forsmark; Adrian D Vickers; Andrew Lloyd
Journal:  Pharmacoeconomics       Date:  2019-06       Impact factor: 4.981

2.  Drug treatment for spinal muscular atrophy types II and III.

Authors:  Renske I Wadman; W Ludo van der Pol; Wendy Mj Bosboom; Fay-Lynn Asselman; Leonard H van den Berg; Susan T Iannaccone; Alexander Fje Vrancken
Journal:  Cochrane Database Syst Rev       Date:  2020-01-06

3.  Novel combinatorial screening identifies neurotrophic factors for selective classes of motor neurons.

Authors:  Sébastien Schaller; Dorothée Buttigieg; Alysson Alory; Arnaud Jacquier; Marc Barad; Mark Merchant; David Gentien; Pierre de la Grange; Georg Haase
Journal:  Proc Natl Acad Sci U S A       Date:  2017-03-07       Impact factor: 11.205

4.  Motor neuron disease: A prospective natural history study of type 1 spinal muscular atrophy.

Authors:  Enrico Bertini; Eugenio Mercuri
Journal:  Nat Rev Neurol       Date:  2018-01-19       Impact factor: 42.937

5.  Spinal Muscular Atrophy Type I: Is It Ethical to Standardize Supportive Care Intervention in Clinical Trials?

Authors:  Richard S Finkel; Kathie M Bishop; Robert M Nelson
Journal:  J Child Neurol       Date:  2016-10-22       Impact factor: 1.987

Review 6.  Spinal muscular atrophy - insights and challenges in the treatment era.

Authors:  Eugenio Mercuri; Maria Carmela Pera; Mariacristina Scoto; Richard Finkel; Francesco Muntoni
Journal:  Nat Rev Neurol       Date:  2020-10-14       Impact factor: 42.937

7.  An observational study of functional abilities in infants, children, and adults with type 1 SMA.

Authors:  Marika Pane; Concetta Palermo; Sonia Messina; Valeria A Sansone; Claudio Bruno; Michela Catteruccia; Maria Sframeli; Emilio Albamonte; Marina Pedemonte; Adele D'Amico; Giorgia Brigati; Roberto de Sanctis; Giorgia Coratti; Simona Lucibello; Enrico Bertini; Giuseppe Vita; Francesco Danilo Tiziano; Eugenio Mercuri
Journal:  Neurology       Date:  2018-07-25       Impact factor: 9.910

8.  Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.

Authors:  Ali Jalali; Erin Rothwell; Jeffrey R Botkin; Rebecca A Anderson; Russell J Butterfield; Richard E Nelson
Journal:  J Pediatr       Date:  2020-07-11       Impact factor: 4.406

9.  Oral and Swallowing Abilities Tool (OrSAT) for Type 1 SMA Patients: Development of a New Module.

Authors:  Beatrice Berti; Lavinia Fanelli; Roberto de Sanctis; Roberta Onesimo; Concetta Palermo; Daniela Leone; Sara Carnicella; Giulia Norcia; Nicola Forcina; Giorgia Coratti; Valentina Giorgio; Antonella Cerchiari; Simona Lucibello; Richard Finkel; Marika Pane; Eugenio Mercuri
Journal:  J Neuromuscul Dis       Date:  2021

10.  Impairment of the neurotrophic signaling hub B-Raf contributes to motoneuron degeneration in spinal muscular atrophy.

Authors:  Niko Hensel; Federica Cieri; Pamela Santonicola; Ines Tapken; Tobias Schüning; Michela Taiana; Elisa Pagliari; Antonia Joseph; Silke Fischer; Natascha Heidrich; Hella Brinkmann; Sabrina Kubinski; Anke K Bergmann; Manuela F Richter; Klaus Jung; Stefania Corti; Elia Di Schiavi; Peter Claus
Journal:  Proc Natl Acad Sci U S A       Date:  2021-05-04       Impact factor: 11.205
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