B
Brenda Wong
Researcher at Cincinnati Children's Hospital Medical Center
Publications - 127
Citations - 5451
Brenda Wong is an academic researcher from Cincinnati Children's Hospital Medical Center. The author has contributed to research in topics: Duchenne muscular dystrophy & Muscular dystrophy. The author has an hindex of 34, co-authored 121 publications receiving 4788 citations. Previous affiliations of Brenda Wong include Boston Children's Hospital & University of Cincinnati.
Papers
More filters
Journal ArticleDOI
Consensus Statement for Standard of Care in Spinal Muscular Atrophy
Ching H. Wang,Richard S. Finkel,Enrico Bertini,Mary K. Schroth,Anita K. Simonds,Brenda Wong,Annie Aloysius,Leslie Morrison,Thomas O. Crawford,Anthony Trela +9 more
TL;DR: It is the authors' intention that this document be used as a guideline, not as a practice standard for spinal muscular atrophy patients, because of the high level of uncertainty in these patients' care.
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.
Journal ArticleDOI
Mutational Spectrum of DMD Mutations in Dystrophinopathy Patients: Application of Modern Diagnostic Techniques to a Large Cohort
Kevin M. Flanigan,D M Dunn,von Niederhausern A,Payam Soltanzadeh,Eduard Gappmaier,Michael T. Howard,Jacinda B. Sampson,Mendell,Cheryl Wall,Wendy King,Alan Pestronk,J. Florence,Anne M. Connolly,Katherine D. Mathews,Stephan Cm,Laubenthal Ks,Laubenthal Ks,Brenda Wong,P. Morehart,Meyer A,Richard S. Finkel,Richard S. Finkel,Carsten G. Bönnemann,Carsten G. Bönnemann,Livija Medne,John W. Day,Joline C. Dalton,Marcia Margolis,Veronica J. Hinton,Robert B. Weiss +29 more
TL;DR: The data supports the uniform hypermutability of CGA>TGA mutations, establishes the frequency of polymorphic muscle (Dp427m) protein isoforms and reveals unique genomic haplotypes associated with “private” mutations.
Journal ArticleDOI
Evolution of the mdx mouse cardiomyopathy: physiological and morphological findings.
TL;DR: Results show that the mdx cardiac function is more impaired than was previously thought and shares important clinical features with the cardiomyopathy of Duchenne muscular dystrophy.