June Brand

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

Eric W.F.W. Alton, Jeffrey Matthijn Beekman, A. Christopher Boyd, June Brand et autres

We have recently shown that non-viral gene therapy can stabilise the decline of lung function in patients with cystic fibrosis (CF). However, the effect was modest, and more potent gene transfer agents are still required. Fuson protein (F)/Hemagglutinin/Neuraminidase protein (HN)-pseudotyped lentiviral vectors …

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Patient information sheets

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Safety and adverse events

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Background Cystic fibrosis (CF) is a chronic, life-limiting disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene leading to abnormal airway surface ion transport, chronic lung infections, inflammation and eventual respiratory failure. With the exception of the small-molecule potentiator, …

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Cystic Fibrosis Questionnaire – Revised quality-of-life questionnaire

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Methods: clinical trial design and outcome measures

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Dose preparation sheet

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Results of mechanistic substudy

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Results of clinical efficacy outcomes

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

Amyloid-β 11 C-PiB-PET imaging results from 2 randomized bapineuzumab phase 3 AD trials

Enchi Liu, Mark E. Schmidt, Richard A. Margolin, Reisa Anne Sperling et autres

OBJECTIVE: To evaluate the effects of bapineuzumab on brain β-amyloid (Aβ) burden using (11)C-Pittsburgh compound B ((11)C-PiB)-PET. METHODS: Two phase 3 clinical trials, 1 each in apolipoprotein APOE ε4 carriers and noncarriers, were conducted in patients with mild to moderate Alzheimer disease …

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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield et autres

BACKGROUND: Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis. METHODS: …

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Assessment of immune responses to single and repeat dose aeroslisation of the non-viral formualtion PGM169/GL67A in cystic fibrosis patients

Uta Griesenbach, Eric W.F.W. Alton, David K Armstrong, Katie J Bayfield et autres