Revised North Star ambulatory assessment for young boys with Duchenne muscular dystrophy
Eugenio Mercuri(Università Cattolica del Sacro Cuore), Marika Pane(Università Cattolica del Sacro Cuore), Adele D’Amico(Bambino Gesù Children's Hospital), Giovanni Baranello, Giuseppe Vita(University of Messina), Giorgia Coratti, Rita De Sanctis(Humanitas University), Sonia Messina(University of Messina), Roberta Battini(Fondazione Stella Maris), Maria Carmela Pera(Università Cattolica del Sacro Cuore), Lavinia Fanelli(Università Cattolica del Sacro Cuore), Enrico Bertini(Bambino Gesù Children's Hospital), Elena Mazzone(Università Cattolica del Sacro Cuore), Emilio Albamonte(University of Milan), Concetta Palermo(Università Cattolica del Sacro Cuore), Serena Sivo(Università Cattolica del Sacro Cuore), Francesco Muntoni(Great Ormond Street Hospital), Valeria Ricotti(Solidus Biosciences (United States))
Cited by 3
Related Papers
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
|New England Journal of Medicine|2018|2.9k
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
|New England Journal of Medicine|2018|1.4k
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
|Science Translational Medicine|2017|809