L
Leslie Raffini
Researcher at Children's Hospital of Philadelphia
Publications - 116
Citations - 5683
Leslie Raffini is an academic researcher from Children's Hospital of Philadelphia. The author has contributed to research in topics: Medicine & Population. The author has an hindex of 32, co-authored 98 publications receiving 4302 citations. Previous affiliations of Leslie Raffini include University of Pennsylvania.
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Journal ArticleDOI
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen R. Russell,Jean Bennett,Jennifer Wellman,Daniel C. Chung,Zi Fan Yu,Amy Tillman,Janet Wittes,Julie Pappas,Okan U. Elci,Sarah McCague,Dominique Cross,Kathleen A. Marshall,Jean Walshire,Taylor Kehoe,Hannah Reichert,Maria C. Davis,Leslie Raffini,Lindsey A. George,F. Parker Hudson,Laura E. Dingfield,Xiaosong Zhu,Julia A. Haller,Elliott H. Sohn,Vinit B. Mahajan,Wanda Pfeifer,Michelle T. Weckmann,Chris A. Johnson,Dina Y. Gewaily,Arlene V. Drack,Edwin M. Stone,Katie Wachtel,Francesca Simonelli,Bart P. Leroy,Bart P. Leroy,J. Fraser Wright,Katherine A. High,Albert M. Maguire +36 more
TL;DR: Voretigene neparvovec gene replacement improved functional vision in RPE65-mediated inherited retinal dystrophy previously medically untreatable.
Journal ArticleDOI
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M. Maguire,Albert M. Maguire,Katherine A. High,Katherine A. High,Alberto Auricchio,J. Fraser Wright,J. Fraser Wright,Eric A. Pierce,Eric A. Pierce,Francesco Testa,Federico Mingozzi,Jeannette L. Bennicelli,Gui-Shuang Ying,Settimio Rossi,Ann Fulton,Kathleen A. Marshall,Sandro Banfi,Daniel C. Chung,Jessica I. W. Morgan,Bernd Hauck,Olga Zelenaia,Xiaosong Zhu,Leslie Raffini,Frauke Coppieters,Elfride De Baere,Kenneth S. Shindler,Nicholas J. Volpe,Enrico Maria Surace,Carmela Acerra,Arkady Lyubarsky,T. Michael Redmond,T. Michael Redmond,Edwin M. Stone,Edwin M. Stone,Junwei Sun,Jenni Fer Uvellman Mcdonnell,Bart P. Leroy,Bart P. Leroy,Francesca Simonelli,Jean Bennett,Jean Bennett +40 more
TL;DR: The safety, extent, and stability of improvement in vision in all patients support the use of AAV-mediated gene therapy for treatment of inherited retinal diseases, with early intervention resulting in the best potential gain.
Journal ArticleDOI
Dramatic increase in venous thromboembolism in children's hospitals in the United States from 2001 to 2007.
TL;DR: This multicenter study demonstrates a dramatic increase in the diagnosis of venous thromboembolism at children's hospitals from 2001 to 2007.
Journal ArticleDOI
Impact of Thrombophilia on Risk of Arterial Ischemic Stroke or Cerebral Sinovenous Thrombosis in Neonates and Children A Systematic Review and Meta-Analysis of Observational Studies
Gili Kenet,Lisa K. Lütkhoff,Manuela Albisetti,Timothy J. Bernard,Mariana Bonduel,Leonardo R. Brandão,Stéphane Chabrier,Anthony K.C. Chan,Gabrielle deVeber,Barbara Fiedler,Heather J. Fullerton,Neil A. Goldenberg,Eric F. Grabowski,Gudrun Günther,Christine Heller,Susanne Holzhauer,Alfonso Iorio,Janna M. Journeycake,Ralf Junker,Fenella J. Kirkham,Fenella J. Kirkham,Karin Kurnik,John K. Lynch,Christoph Male,Marilyn J. Manco-Johnson,Rolf M. Mesters,Paul Monagle,C. Heleen van Ommen,Leslie Raffini,Kevin Rostasy,Paolo Simioni,Ronald Sträter,Guy Young,Ulrike Nowak-Göttl +33 more
TL;DR: It is indicated that thrombophilias serve as risk factors for incident stroke through a meta-analysis of published observational studies and no significant heterogeneity was discerned across studies, and no publication bias was detected.
Journal ArticleDOI
American Society of Hematology 2018 Guidelines for management of venous thromboembolism: treatment of pediatric venous thromboembolism.
Paul Monagle,Carlos Cuello,Carlos Cuello,Caitlin Augustine,Mariana Bonduel,Leonardo R. Brandão,Tammy Capman,Anthony K.C. Chan,Sheila J. Hanson,Christoph Male,Joerg J Meerpohl,Fiona Newall,Sarah H. O'Brien,Leslie Raffini,Heleen van Ommen,John Wiernikowski,Suzan Williams,Meha Bhatt,John J. Riva,Yetiani Roldan,Nicole Schwab,Reem A. Mustafa,Reem A. Mustafa,Sara K. Vesely +23 more
TL;DR: Recommendations include understanding the natural history of asymptomatic thrombosis, determining subgroup boundaries that enable risk stratification of children for escalation of treatment, and appropriate study of newer anticoagulant agents in children.