G
G. Campion
Researcher at BioMarin Pharmaceutical
Publications - 32
Citations - 1560
G. Campion is an academic researcher from BioMarin Pharmaceutical. The author has contributed to research in topics: Duchenne muscular dystrophy & Drisapersen. The author has an hindex of 12, co-authored 28 publications receiving 1312 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
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.
Anna Mayhew,Elena S. Mazzone,Michelle Eagle,T. Duong,Maria Ash,Valérie Decostre,Marlene Vandenhauwe,Katrijn Klingels,Julaine Florence,Marion Main,Flaviana Bianco,Erik Henrikson,Laurent Servais,G. Campion,Elizabeth Vroom,Valeria Ricotti,Natalie Goemans,Craig M. McDonald,Eugenio Mercuri +18 more
TL;DR: The steps leading to the development of the Performance of the Upper Limb (PUL), a tool specifically designed for assessing upper limb function in ambulant and non‐ambulant patients with Duchenne muscular dystrophy (DMD), are reported.
Journal ArticleDOI
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.
Nathalie Goemans,Eugenio Mercuri,Elena Belousova,Hirofumi Komaki,Alberto Dubrovsky,Craig M. McDonald,John E. Kraus,Afrodite Lourbakos,Zhengning Lin,G. Campion,S.X. Wang,Craig Campbell +11 more
TL;DR: It is suggested that drisapersen could have benefit in a less impaired population of DMD subjects and the statistical power from pre-specified 90% to actual 53%.
Journal ArticleDOI
Single Ascending Dose Study of a Short Interfering RNA Targeting Lipoprotein(a) Production in Individuals With Elevated Plasma Lipoprotein(a) Levels.
Steven E. Nissen,Kathy Wolski,Craig Balog,Daniel I. Swerdlow,Alison Scrimgeour,Curtis Rambaran,Rosamund Wilson,Malcom Boyce,Kausik K. Ray,Leslie Cho,Gerald F. Watts,Michael J. Koren,Traci Turner,Erik S.G. Stroes,Carrie Melgaard,G. Campion +15 more
TL;DR: In this phase 1 study of 32 participants with elevated Lp(a) levels and no known cardiovascular disease, the siRNA SLN360 was well tolerated, and a dose-dependent lowering of plasma Lp (a) concentrations was observed.
Journal ArticleDOI
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
Volker Straub,Pavel Balabanov,Kate Bushby,Monica Ensini,Nathalie Goemans,Annamaria De Luca,Alejandra Pereda,Robert Hemmings,G. Campion,Edward M. Kaye,Virginia Arechavala-Gomeza,Aurélie Goyenvalle,Erik H. Niks,O. Veldhuizen,Pat Furlong,Violeta Stoyanova-Beninska,Matthew J.A. Wood,A. Johnson,Eugenio Mercuri,Francesco Muntoni,Bruno Sepodes,Manuel Haas,Elizabeth Vroom,Annemieke Aartsma-Rus,Annemieke Aartsma-Rus +24 more
TL;DR: An open and constructive dialogue among European stakeholders has positively affected development of treatments for Duchenne muscular dystrophy; this approach could serve as a paradigm for development of treatment for rare diseases in general.