L
Lia Liefaard
Researcher at GlaxoSmithKline
Publications - 15
Citations - 539
Lia Liefaard is an academic researcher from GlaxoSmithKline. The author has contributed to research in topics: Placebo & Population. The author has an hindex of 6, co-authored 12 publications receiving 462 citations. Previous affiliations of Lia Liefaard include University of Hertfordshire.
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Journal ArticleDOI
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
Thomas Voit,Haluk Topaloglu,Volker Straub,Francesco Muntoni,Nicolas Deconinck,Giles G Campion,Sjef J. de Kimpe,Michelle Eagle,Michela Guglieri,Steve S Hood,Lia Liefaard,Afrodite Lourbakos,Allison A Morgan,Joanna Nakielny,Naashika Quarcoo,Valeria Ricotti,Katie Rolfe,Laurent Servais,C. Wardell,Rosamund Wilson,Padraig Wright,Padraig Wright,John E. Kraus +22 more
TL;DR: Continuous drisapersen resulted in some benefit in 6MWD versus placebo at week 25 in patients in the intention-to-treat population for whom data were available, andAmbulation improvements in this young population with early-stage Duchenne muscular dystrophy are encouraging but need to be confirmed in larger studies.
Journal ArticleDOI
Repeat doses of antibody to serum amyloid P component clear amyloid deposits in patients with systemic amyloidosis
Duncan Richards,Louise M. Cookson,Sharon V. Barton,Lia Liefaard,Thirusha Lane,David F. Hutt,JM Ritter,Marianna Fontana,James C. Moon,Julian D. Gillmore,Ashutosh D. Wechalekar,Philip N. Hawkins,Mark B. Pepys +12 more
TL;DR: It is shown that previous depletion of circulating SAP by the drug, miridesap, uniquely enables subsequent administration of the humanized anti-SAP antibody, dezamizumab, to patients with systemic amyloidosis.
Journal ArticleDOI
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial
Kevin M. Flanigan,Thomas Voit,Xiomara Q. Rosales,Laurent Servais,John E. Kraus,C. Wardell,A. Morgan,Susie Dorricott,Joanna Nakielny,Naashika Quarcoo,Lia Liefaard,Tom Drury,G. Campion,Padraig Wright +13 more
TL;DR: Safety, tolerability, and pharmacokinetics of drisapersen after a single subcutaneous administration in non-ambulatory subjects were assessed; however, at the 9 mg/kg dose, pyrexia and transient elevations in inflammatory parameters were seen.
Journal ArticleDOI
Effects of a novel sodium channel blocker, GSK2339345, in patients with refractory chronic cough .
Jaclyn A. Smith,Lorcan McGarvey,Huda Badri,Imran Satia,Francis Warren,Sarah Siederer,Lia Liefaard,Robert D. Murdoch,Kathryn Povey,Joanna Marks-Konczalik +9 more
TL;DR: It is strongly suggested that GSK2339345 has no anti-tussive effect despite reaching airway sensory nerves as evidenced by the evoked transient cough.
Journal ArticleDOI
The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice.
Ingrid E.C. Verhaart,Laura van Vliet-van den Dool,Jessica A. Sipkens,Sjef J. de Kimpe,Ingrid G.M. Kolfschoten,Judith C.T. van Deutekom,Lia Liefaard,Jim Ridings,Steve R. Hood,Annemieke Aartsma-Rus +9 more
TL;DR: Understanding of the pharmacokinetics and pharmacodynamics of 2′-O-methyl phosphorothioate oligos used for the treatment of DMD is enhanced and Concordance between levels of oligonucleotides, exon skipping, and proteins was observed, except in heart.