Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study

Author:

Messina Sonia,Vita Gian Luca,Sframeli Maria,Mondello Stefania,Mazzone Elena,D'Amico Adele,Berardinelli Angela,La Rosa Matteo,Bruno Claudio,Distefano Maria Grazia,Baranello Giovanni,Barcellona Costanza,Scutifero Marianna,Marcato Sonia,Palmieri Arianna,Politano Luisa,Morandi Lucia,Mongini Tiziana,Pegoraro Elena,D'Angelo Maria Grazia,Pane Marika,Rodolico Carmelo,Minetti Carlo,Bertini Enrico,Vita Giuseppe,Mercuri Eugenio

Funder

Telethon UILDM

Publisher

Elsevier BV

Subject

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

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4. Gene therapy for muscular dystrophy: moving the field forward;Al-Zaidy;Pediatr Neurol,2014

5. Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases;Veltrop;Exp Cell Res,2014

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