Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Autor: Nicolas Deconinck, Steve S Hood, Haluk Topaloglu, Lia Liefaard, C. Wardell, Rosamund Wilson, Sjef J. de Kimpe, John E. Kraus, Michelle Eagle, Katie Rolfe, Laurent Servais, Joanna Nakielny, Afrodite Lourbakos, Michela Guglieri, Valeria Ricotti, Giles G Campion, Allison A Morgan, Francesco Muntoni, Volker Straub, Padraig Wright, Thomas Voit, Naashika Quarcoo

Publikováno v: The Lancet Neurology. 13:987-996

Summary Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We invest

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4376b8b1e340ad1c94d88d59dcefb3a3
https://doi.org/10.1016/s1474-4422(14)70195-4

Quality standards for the management of alcohol-related liver disease: consensus recommendations from the British Association for the Study of the Liver and British Society of Gastroenterology ARLD special interest group.

Autor: Parker R; Leeds Liver Unit, Leeds Teaching Hospitals NHS Trust, Leeds, UK richardparker@nhs.net.; Leeds Institute of Medical Research at St James's University Hospital, University of Leeds, Leeds, UK., Allison M; Cambridge Liver Unit, Addenbrooke's Hospital, Cambridge, UK., Anderson S; Angus Integrated Drug and Alcohol Recovery Service (AIDARS), Ninewells Hospital and Medical School, Dundee, UK., Aspinall R; Gastroenterology & Hepatology, Portsmouth Hospitals NHS Trust, Portsmouth, UK., Bardell S; Birmingham Liver Services Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Bains V; Liver Transplant Unit, King's College Hospital NHS Foundation Trust, London, UK., Buchanan R; Faculty of Medicine, University of Southampton, Southampton, UK., Corless L; Department of Gastroenterology, Hepatology and Endoscopy, Hull University Teaching Hospitals NHS Trust, Hull, UK., Davidson I; NHS Fife Addiction Services, NHS Fife, Kirkcaldy, UK., Dundas P; Peter Brunt Centre, Aberdeen Royal Infirmary, NHS Grampian, Aberdeen, UK., Fernandez J; Alcohol and Drug Liaison, Royal Free London NHS Foundation Trust, London, UK., Forrest E; Dept of Gastroenterology, Glasgow Royal Infirmary, Glasgow, UK., Forster E; Leeds Liver Unit, Leeds Teaching Hospitals NHS Trust, Leeds, UK., Freshwater D; Birmingham Liver Services Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Gailer R; Islington Primary Care Federation, London, UK., Goldin R; Department of Digestive Diseases, Department of Medicine, Imperial College London, London, UK., Hebditch V; British Liver Trust, Ringwood, UK., Hood S; Digestive Diseases Unit, Liverpool University Hospitals NHS Foundation Trust, Liverpool, UK., Jones A; Pharmacy, Barts and The London NHS Trust, London, UK., Lavers V; Independent researcher, Plymouth, UK., Lindsay D; Alcohol Care Team, East Lancashire Hospitals NHS Trust, Blackburn, UK., Maurice J; Gastroenterology and hepatology, North Bristol NHS Trust, Westbury on Trym, UK., McDonagh J; Birmingham Liver Services Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Morgan S; Hampstead Group Practice, London, UK., Nurun T; Department of Gastroenterology, Hepatology and Endoscopy, Hull University Teaching Hospitals NHS Trust, Hull, UK., Oldroyd C; Cambridge Liver Unit, Addenbrooke's Hospital, Cambridge, UK., Oxley E; St Martins Practice, Leeds, UK., Pannifex S; Hepatology, St George's Healthcare NHS Trust, London, UK., Parsons G; Hepatology, Plymouth Hospitals NHS Trust, Plymouth, UK., Phillips T; Faculty of Health Sciences, University of Hull, Hull, UK., Rainford N; Liver Transplant Unit, King's College Hospital NHS Foundation Trust, London, UK., Rajoriya N; Birmingham Liver Services Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Richardson P; Gastroenterology and Hepatology, Royal Liverpool and Broadgreen University Hospitals NHS Trust, Liverpool, UK., Ryan J; Sheila Sherlock Liver Centre, Royal Free London NHS Foundation Trust, London, UK., Sayer J; Gastroenterology, Doncaster and Bassetlaw Hospitals NHS Foundation Trust, Doncaster, UK., Smith M; Alcohol care team, Southport and Ormskirk Hospital NHS Trust, Southport, UK., Srivastava A; Gastroenterology and hepatology, North Bristol NHS Trust, Westbury on Trym, UK., Stennett E; Gastroenterology, Guy's and St Thomas' Hospitals NHS Trust, London, UK., Towey J; Birmingham Liver Services Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Vaziri R; Humankind, Durham, UK., Webzell I; Liver Transplant Unit, King's College Hospital NHS Foundation Trust, London, UK., Wellstead A; Gastroenterology, University Hospitals Dorset NHS Foundation Trust, Poole, UK., Dhanda A; Faculty of health, University of Plymouth, Plymouth, UK., Masson S; Liver unit, The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle, UK.

Publikováno v: BMJ open gastroenterology [BMJ Open Gastroenterol] 2023 Oct; Vol. 10 (1).

Quantification of Drugs in Brain and Liver Mimetic Tissue Models Using Raman Spectroscopy.

Autor: Woodhouse N; School of Physics and Astronomy, 6123University of Nottingham, Nottingham, UK., Majer J; 113460GlaxoSmithKline, Stevenage, UK., Marshall P; 113460GlaxoSmithKline, Stevenage, UK., Hood S; 113460GlaxoSmithKline, Stevenage, UK., Notingher I; School of Physics and Astronomy, 6123University of Nottingham, Nottingham, UK.

Publikováno v: Applied spectroscopy [Appl Spectrosc] 2023 Mar; Vol. 77 (3), pp. 246-260. Date of Electronic Publication: 2022 Nov 16.

Comparison of Pharmacokinetics of the GalNAc-Conjugated Antisense Oligonucleotide GSK3389404 in Participants with Chronic Hepatitis B Infection across the Asia-Pacific Region.

Autor: Han K; GSK, Collegeville, Pennsylvania, USA., Ito H; GSK K.K., Tokyo, Japan., Elston R; GSK, Stevenage, United Kingdom., Cremer J; GSK, Durham, North Carolina, USA., Hood S; GSK, Stevenage, United Kingdom., Paff M; GSK, Collegeville, Pennsylvania, USA., Theodore D; GSK, Durham, North Carolina, USA.

Publikováno v: Antimicrobial agents and chemotherapy [Antimicrob Agents Chemother] 2023 Jan 24; Vol. 67 (1), pp. e0090022. Date of Electronic Publication: 2022 Dec 12.

Meeting report: oligonucleotide ADME workshop.

Autor: Hood S; Department of Research, In Vitro/In Vivo Translation, GlaxoSmithKline, Stevenage, UK., Kenworthy D; Department of Research, In Vitro/In Vivo Translation, GlaxoSmithKline, Stevenage, UK., Christensen JK; Development ADME, Novo Nordisk A/S, Måløv, Denmark.

Publikováno v: Xenobiotica; the fate of foreign compounds in biological systems [Xenobiotica] 2022 Aug; Vol. 52 (8), pp. 957-961. Date of Electronic Publication: 2022 Sep 12.