Nusinersen in type 1 SMA infants, children and young adults: Preliminary results on motor function-Reference-Cited by-同舟云学术

Nusinersen in type 1 SMA infants, children and young adults: Preliminary results on motor function

Published:2018-07 Issue:7 Volume:28 Page:582-585
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Pane Marika,Palermo Concetta,Messina Sonia,Sansone Valeria A,Bruno Claudio,Catteruccia Michela,Sframeli Maria,Albamonte Emilio,Pedemonte Marina,D'Amico Adele,Brigati Giorgia,de Sanctis Roberto,Coratti Giorgia,Lucibello Simona^ORCID,Bertini Enrico^ORCID,Vita Giuseppe,Tiziano Francesco Danilo^ORCID,Mercuri Eugenio

Publisher

Elsevier BV

Subject

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

Reference14 articles.

1. Nusinersen versus sham control in infantile-onset spinal muscular atrophy;Finkel;N Engl J Med,2017

2. Nusinersen versus sham control in later-onset spinal muscular atrophy;Mercuri;N Engl J Med,2018

3. Expanded access program with Nusinersen in SMA type I in Italy: strengths and pitfalls of a successful experience;Messina;Neuromusc Disord,2017

4. Single-dose gene-replacement therapy for spinal muscular atrophy;Mendell;N Engl J Med,2017

5. The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability;Glanzman;Neuromusc Disord,2010

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