Diagnosis and Management of Type 1 Sialidosis: Clinical Insights from Long-Term Care of Four Unrelated Patients
Antonietta Coppola(University of Naples Federico II), Leonilda Bilo(University of Naples Federico II), Alessandra D’Amico(Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico), Lorenzo Ugga(University of Campania "Luigi Vanvitelli"), Barbara Castellotti(Fondazione IRCCS Istituto Neurologico Carlo Besta), Serena Troisi(Federico II University Hospital), Marta Ianniciello(Federico II University Hospital), Ebru Nur Vanlı-Yavuz(Koç University), Marcello Esposito(Ospedale Antonio Cardarelli), Marta Bellofatto(Fondazione IRCCS Istituto Neurologico Carlo Besta), Betül Baykan(Istanbul Eye Hospital), Pasquale Striano(Great Ormond Street Hospital), Settimio Rossi(University of Naples Federico II), Stefano Tozza(University of Naples Federico II), Salvatore Striano(Federico II University Hospital), Francesca Simonelli(University of Naples Federico II)
Cited by 19
Related Papers
Mapping the human genetic architecture of COVID-19
|Nature|2021|1.1k
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
|The Lancet|2009|857
Recurrent Rearrangements of Chromosome 1q21.1 and Variable Pediatric Phenotypes
|New England Journal of Medicine|2008|811
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
|Molecular Therapy|2009|551
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
|Science Translational Medicine|2012|390