C
Cas Donoghue
Researcher at Nationwide Children's Hospital
Publications - 6
Citations - 590
Cas Donoghue is an academic researcher from Nationwide Children's Hospital. The author has contributed to research in topics: Eteplirsen & Duchenne muscular dystrophy. The author has an hindex of 3, co-authored 6 publications receiving 477 citations.
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Journal Article
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy (DMD) (S42.001)
Jay S. Charleston,F.J. Schnell,Johannes Dworzak,Cas Donoghue,J Lynch,Sarah Lewis,Lei Chen,Louise R. Rodino-Klapac,Zarife Sahenk,Jon Voss,U. DeAlwis,D. Frank,H Eliopoulos,J. Mendell +13 more
TL;DR: The present study used a double‐blind placebo‐controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6‐minute walk test.
Journal ArticleDOI
Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production.
Jay S. Charleston,Frederick J. Schnell,Johannes Dworzak,Cas Donoghue,Sarah Lewis,Lei Chen,G. David Young,Anthony J. Milici,Jon Voss,Uditha DeAlwis,Bruce M. Wentworth,Louise R. Rodino-Klapac,Zarife Sahenk,D. Frank,Jerry R. Mendell +14 more
TL;DR: This study provides Class II evidence of the muscle cell penetration, exon skipping, and induction of novel dystrophin expression by eteplirsen, as confirmed by 4 assays.
Journal ArticleDOI
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy.
Lindsay N. Alfano,Jay S. Charleston,Anne M. Connolly,Anne M. Connolly,Linda H. Cripe,Cas Donoghue,Robert Dracker,Johannes Dworzak,Helen Eliopoulos,D. Frank,Sarah Lewis,Karin Lucas,Jessie Lynch,Anthony J. Milici,Amy Flynt,Emily Naughton,Louise R. Rodino-Klapac,Zarife Sahenk,Frederick J. Schnell,G. David Young,Jerry R. Mendell,Linda Lowes +21 more
TL;DR: Despite the loss of ambulation, other markers of disease progression remained relatively stable in the eteplirsen-treated twin patients and were similar to those of the ambulatory patients.
Journal Article
Development of a validated Western blot method for quantification of human dystrophin protein used in Phase II and III clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) (P5.105)
F.J. Schnell,Cas Donoghue,Johannes Dworzak,Jay S. Charleston,D. Frank,Steve D. Wilton,Sarah Lewis,J. Mendell,Louise R. Rodino-Klapac,Zarife Sahenk +9 more
TL;DR: The Western blot method was validated according to FDA Draft Guidance for Industry, Bioanalytical Method Validation, and can provide confirmation of mechanism of action for dystrophin restoring therapies.
Journal Article
Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for Duchenne Muscular Dystrophy (DMD): Clinical Update and Longitudinal Comparison to External Controls on Six-Minute Walk Test (6MWT) (S28.001)
J. Mendell,Nathalie Goemans,Louise R. Rodino-Klapac,Zarife Sahenk,Linda Lowes,Lindsay N. Alfano,K. Berry,E. Peterson,Sarah Lewis,Kim Shontz,J. Shao,Petra W. Duda,Cas Donoghue,F.J. Schnell,Johannes Dworzak,Bruce M. Wentworth,Edward M. Kaye,Eugenio Mercuri +17 more
TL;DR: After 3 years of eteplirsen-treatment, DMD patients had a mean 6MWT that was 151m higher than the national average, andWestern blot confirmed dystrophin production in 9/11 patients.