N
Niki Heuvelmans
Publications - 3
Citations - 1019
Niki Heuvelmans is an academic researcher. The author has contributed to research in topics: Duchenne muscular dystrophy & Dystrophin. The author has an hindex of 3, co-authored 3 publications receiving 987 citations.
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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
In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.
Hans Heemskerk,Christa L. de Winter,Sjef J. de Kimpe,Petra Van Kuik-Romeijn,Niki Heuvelmans,Gerard Johannes Platenburg,Gert-Jan B. van Ommen,Judith C.T. van Deutekom,Annemieke Aartsma-Rus +8 more
TL;DR: Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne into a milder Becker muscular dystrophy phenotype.
Journal ArticleDOI
Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
Hans Heemskerk,Christa L. de Winter,Petra Van Kuik,Niki Heuvelmans,Patrizia Sabatelli,Paola Rimessi,Paola Braghetta,Gert-Jan B. van Ommen,Sjef J. de Kimpe,Alessandra Ferlini,Annemieke Aartsma-Rus,Judith C.T. van Deutekom +11 more
TL;DR: The results demonstrate that the levels of oligonucleotides in dystrophin-deficient muscle fibers are much higher than in healthy fibers, leading to higher exon-skipping levels, and this patient-convenient delivery method is selected for future clinical study protocols.