N
Nathalie Goemans
Researcher at Katholieke Universiteit Leuven
Publications - 162
Citations - 7565
Nathalie Goemans is an academic researcher from Katholieke Universiteit Leuven. The author has contributed to research in topics: Duchenne muscular dystrophy & Medicine. The author has an hindex of 37, co-authored 142 publications receiving 6178 citations. Previous affiliations of Nathalie Goemans include Boston Children's Hospital & Flanders Institute for Biotechnology.
Papers
More filters
Journal ArticleDOI
Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
Judith C.T. van Deutekom,Anneke A.M. Janson,Ieke B. Ginjaar,Wendy S. Frankhuizen,Annemieke Aartsma-Rus,Mattie Bremmer-Bout,Johan T. den Dunnen,Klaas Koop,Anneke J. van der Kooi,Nathalie Goemans,Sjef J. de Kimpe,Peter F. Ekhart,Edna H. Venneker,Gerard Johannes Platenburg,Jan J.G.M. Verschuuren,Gert-Jan B. van Ommen +15 more
TL;DR: Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations, suggesting that further studies might be feasible.
Journal ArticleDOI
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
Nathalie Goemans,Mar Tulinius,Johanna T van den Akker,Brigitte E Burm,Peter F. Ekhart,Niki Heuvelmans,Tjadine Holling,Anneke A.M. Janson,Gerard Johannes Platenburg,Jessica A. Sipkens,J M Ad Sitsen,Annemieke Aartsma-Rus,Gert-Jan B. van Ommen,Gunnar Buyse,Niklas Darin,Jan J.G.M. Verschuuren,G. Campion,Sjef J. de Kimpe,Judith C.T. van Deutekom +18 more
TL;DR: Systemically administered PRO051 showed dose-dependent molecular efficacy in patients with Duchenne's muscular dystrophy, with a modest improvement in the 6-minute walk test after 12 weeks of extended treatment.
Journal ArticleDOI
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
Jerry R. Mendell,Nathalie Goemans,Linda Lowes,Lindsay N. Alfano,K. Berry,James Shao,Edward M. Kaye,Eugenio Mercuri +7 more
TL;DR: In this article, the authors evaluated the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip exon 51 in patients with Duchenne muscular dystrophy.
Journal ArticleDOI
MFN2 mutation distribution and genotype/phenotype correlation in Charcot-Marie-Tooth type 2.
Kristien Verhoeven,Kristl Claeys,Stephan Züchner,Stephan Züchner,J. Michael Schröder,Joachim Weis,Chantal Ceuterick,Albena Jordanova,Eva Nelis,Els De Vriendt,Matthias Van Hul,Pavel Seeman,Radim Mazanec,Gulam Mustafa Saifi,Kinga Szigeti,Pedro Mancias,Ian J. Butler,Andrzej Kochański,Barbara Ryniewicz,Jan De Bleecker,Peter Van den Bergh,Christine Verellen,Rudy Van Coster,Nathalie Goemans,Michaela Auer-Grumbach,Wim Robberecht,Vedrana Milic Rasic,Yoram Nevo,I. Tournev,Velina Guergueltcheva,Filip Roelens,Peter Vieregge,Paolo Vinci,María Teresa García Moreno,H.-J. Christen,Michael E. Shy,James R. Lupski,Jeffery M. Vance,Peter De Jonghe,Vincent Timmerman +39 more
TL;DR: Electrophysiological data showed in the majority of patients normal to slightly reduced nerve conduction velocities with often severely reduced amplitudes of the compound motor and sensory nerve action potentials, whereas a smaller group experienced a later onset and milder disease course.
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.