M
Marcio Souza
Researcher at Shire plc
Publications - 11
Citations - 481
Marcio Souza is an academic researcher from Shire plc. The author has contributed to research in topics: Ataluren & Duchenne muscular dystrophy. The author has an hindex of 6, co-authored 10 publications receiving 318 citations.
Papers
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Journal ArticleDOI
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Craig M. McDonald,Craig Campbell,Ricardo Erazo Torricelli,Richard S. Finkel,Richard S. Finkel,Kevin M. Flanigan,Nathalie Goemans,Peter Heydemann,Anna Kamińska,Janbernd Kirschner,Francesco Muntoni,Andrés Nascimento Osorio,Ulrike Schara,Thomas Sejersen,Perry B. Shieh,H. Lee Sweeney,Haluk Topaloglu,Mar Tulinius,Juan J. Vílchez,Thomas Voit,Thomas Voit,Brenda Wong,Gary Elfring,H. Kroger,Xiaohui Luo,Joseph McIntosh,Tuyen Ong,Peter Riebling,Marcio Souza,R. Spiegel,Stuart W. Peltz,Eugenio Mercuri,Lindsay N. Alfano,Michelle Eagle,M. James,Linda Lowes,Anna Mayhew,Elena S. Mazzone,Leslie Nelson,Kristy Rose,Hoda Abdel-Hamid,Susan D. Apkon,Richard J. Barohn,Enrico Bertini,Clemens Bloetzer,Lausanne Canton de Vaud,Russell J. Butterfield,Brigitte Chabrol,Jong-Hee Chae,Daehak-ro Jongno-gu,Giacomi Pietro Comi,Basil T. Darras,Jahannaz Dastgir,Isabelle Desguerre,Raul G Escobar,Erika Finanger,Michela Guglieri,Imelda Hughes,Susan T. Iannaccone,Kristi J. Jones,Peter I. Karachunski,Martin Kudr,Timothy Lotze,Jean K. Mah,Katherine D. Mathews,Yoram Nevo,Julie A. Parsons,Yann Péréon,Alexandra Prufer de Queiroz Campos Araujo,J. Ben Renfroe,Maria Bernadete Dutra de Resende,Monique M. Ryan,Kathryn Selby,Gihan Tennekoon,Giuseppe Vita +74 more
TL;DR: Ataluren was generally well tolerated and most treatment-emergent adverse events were mild to moderate in severity, and there was a significant effect of ataluren in the prespecified subgroup of patients in the intention-to-treat population.
Journal ArticleDOI
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials
Craig M. McDonald,Gautam Sajeev,Zhiwen Yao,Erin McDonnell,Gary Elfring,Marcio Souza,Stuart W. Peltz,Basil T. Darras,Perry B. Shieh,David A. Cox,John Landry,James Signorovitch +11 more
TL;DR: In this study, disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy clinical trials is characterized.
Journal ArticleDOI
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
Perry B. Shieh,Joseph McIntosh,Fengbin Jin,Marcio Souza,Gary Elfring,Siva Narayanan,Panayiota Trifillis,Stuart W. Peltz,Craig M. McDonald,Basil T. Darras +9 more
TL;DR: This analysis suggests greater preservation of 6MWD and 4‐stair climb with deflazacort vs. prednisone/prednisolone in the placebo arm, and a head‐to‐head comparison will better define these differences.
Journal ArticleDOI
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
Craig Campbell,Richard J. Barohn,Enrico Bertini,Brigitte Chabrol,Giacomo P. Comi,Basil T. Darras,Richard S. Finkel,Kevin M. Flanigan,Nathalie Goemans,Susan T. Iannaccone,Kristi J. Jones,Janbernd Kirschner,Jean K. Mah,Katherine D. Mathews,Craig M. McDonald,Eugenio Mercuri,Yoram Nevo,Yann Péreón,J. Ben Renfroe,Monique M. Ryan,Jacinda B. Sampson,Ulrike Schara,Thomas Sejersen,Kathryn Selby,Mar Tulinius,Juan J. Vílchez,Thomas Voit,Lee-Jen Wei,Brenda L. Wong,Gary Elfring,Marcio Souza,Joseph McIntosh,Panayiota Trifillis,Stuart W. Peltz,Francesco Muntoni +34 more
TL;DR: Meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks, and treatment benefit was most evident in Patients with a baseline 6MWD ≥300-<400 m (the ambulatory transition phase), thereby informing future trial design.
Patent
Systems and methods for managing treatment of an orphan disease
TL;DR: In this paper, a system for managing treatment of an orphan disease of a patient by a user includes a mobile device and a program stored thereon, which is constructed and adapted to communicate with at least one server.