K
Kevin M. Flanigan
Researcher at Nationwide Children's Hospital
Publications - 74
Citations - 4450
Kevin M. Flanigan is an academic researcher from Nationwide Children's Hospital. The author has contributed to research in topics: Duchenne muscular dystrophy & Muscular dystrophy. The author has an hindex of 23, co-authored 74 publications receiving 3641 citations. Previous affiliations of Kevin M. Flanigan include The Research Institute at Nationwide Children's Hospital & The Ohio State University Wexner Medical Center.
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Journal ArticleDOI
The muscular dystrophies.
TL;DR: Advances in the field include improved methods of diagnosis, continued identification of disease genes, and the development of a unified model of pathogenesis in facioscapulohumeral dystrophy, which are reflected in thedevelopment of new therapeutic approaches.
Journal ArticleDOI
Evidence-based path to newborn screening for Duchenne muscular dystrophy.
Jerry R. Mendell,Chris Shilling,Nancy D. Leslie,Kevin M. Flanigan,Roula al-Dahhak,Julie M. Gastier-Foster,Kelley Kneile,Diane M. Dunn,Brett Duval,Alexander Aoyagi,Cindy Hamil,Maha Mahmoud,Kandice Roush,Lauren Bird,Chelsea Rankin,Heather Lilly,Natalie Street,Ram Chandrasekar,Robert B. Weiss +18 more
TL;DR: A 2‐tier system using the dried blood spot to first assess CK with follow‐up DMD gene testing is introduced to assess CK in Duchenne muscular dystrophy.
Journal ArticleDOI
The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
Catherine L. Bladen,David Salgado,Soledad Monges,Maria E. Foncuberta,Kyriaki Kekou,Konstantina Kosma,Konstantina Kosma,Hugh Dawkins,Leanne Lamont,Anna J. Roy,Teodora Chamova,Velina Guergueltcheva,Sophelia H. S. Chan,Lawrence Korngut,Craig Campbell,Yi Dai,Jen Wang,Nina Barišić,Petr Brabec,Jaana Lahdetie,Maggie C. Walter,Olivia Schreiber-Katz,Veronika Karcagi,Marta Garami,Venkatarman Viswanathan,Farhad Bayat,Filippo Buccella,En Kimura,Zaïda Koeks,Janneke C. van den Bergen,Miriam Rodrigues,Richard Roxburgh,Anna Lusakowska,Anna Kostera-Pruszczyk,Janusz Zimowski,Rosário Santos,Elena Neagu,Svetlana Artemieva,Vedrana Milic Rasic,Dina Vojinovic,Manuel Posada,Clemens Bloetzer,Pierre Yves Jeannet,Franziska Joncourt,Jordi Díaz-Manera,Eduard Gallardo,Ayse Karaduman,Haluk Topaloglu,Rasha El Sherif,Angela Stringer,Andriy V. Shatillo,Ann Martin,Holly L. Peay,Matthew I. Bellgard,Jan Kirschner,Kevin M. Flanigan,Volker Straub,Kate Bushby,Jan J.G.M. Verschuuren,Annemieke Aartsma-Rus,Annemieke Aartsma-Rus,Christophe Béroud,Christophe Béroud,Hanns Lochmüller +63 more
TL;DR: The development and analysis of the TREAT‐NMD DMD Global database is described and mutations were identified that would potentially benefit from novel genetic therapies for DMD including stop codon read‐through therapies and exon skipping therapy.
Journal ArticleDOI
Ataluren treatment of patients with nonsense mutation dystrophinopathy
Katharine Bushby,Richard S. Finkel,Brenda Wong,Richard J. Barohn,Craig Campbell,Giacomo P. Comi,Anne M. Connolly,John W. Day,Kevin M. Flanigan,Nathalie Goemans,Kristi J. Jones,Eugenio Mercuri,Ros Quinlivan,James B. Renfroe,Barry S. Russman,Monique M. Ryan,Mar Tulinius,Thomas Voit,Steven A. Moore,H. Lee Sweeney,Richard T. Abresch,Kim L. Coleman,Michelle Eagle,Julaine Florence,Eduard Gappmaier,Allan M. Glanzman,Erik K Henricson,Jay A. Barth,Gary Elfring,A. Reha,R. Spiegel,Michael W. O'donnell,Stuart W. Peltz,Craig M. McDonald +33 more
TL;DR: As the first investigational new drug targeting the underlying cause of nm‐dystrophinopathy, ataluren offers promise as a treatment for this orphan genetic disorder with high unmet medical need.
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.