The Spinal Muscular Atrophy Health Index: Italian validation of a disease-specific outcome measure

Author:

Sansone Valeria A.,Pirola Alice,Lizio Andrea,Greco Lucia Catherine,Coratti Giorgia,Casiraghi Jacopo,Pane Marika,Pera Maria Carmela,Italiano Cristina,Messina Sonia,Pozzi Susanna,Sframeli Maria,D'Amico Adele,Bertini Enrico,Bruno Claudio,Mauro Luca,Salmin Francesca,Stancanelli Claudia,Pedemonte Marina,Albamonte Emilio,Zizzi Christine,Heatwole Chad,Mercuri Eugenio

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference20 articles.

1. Spinal muscular atrophy;Lunn;Lancet,2008

2. Childhood spinal muscular atrophy: controversies and challenges;Mercuri;Lancet Neurol,2012

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4. Discrepancy in redetermination of SMN2 copy numbers in children with SMA;Schorling;Neurology,2019

5. Natural history data in adults with SMA;Vázquez-Costa;Lancet Neurol,2020

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