Systematic review of N-of-1 studies in rare genetic neurodevelopmental disorders: the power of 1

AR Müller, MMMG Brands, PM van de Ven, KCB Roes… - Neurology, 2021 - neurology.org
Objective To improve the use of N-of-1 studies in rare genetic neurodevelopmental
disorders, we systematically reviewed the literature and formulated recommendations for …

So near yet so far: why won't the UK prescribe medical cannabis?

D Nutt, S Bazire, LD Phillips, AK Schlag - BMJ open, 2020 - bmjopen.bmj.com
Although cannabis-based products for medicinal use are now legal in the UK, it is still
challenging for patients to gain access, and only very few National Health Service …

[HTML][HTML] Methods used to develop the SPIRIT 2024 and CONSORT 2024 Statements

R Tunn, I Boutron, AW Chan, GS Collins… - Journal of Clinical …, 2024 - Elsevier
Objectives To describe, and explain the rationale for, the methods used and decisions made
during development of the updated SPIRIT 2024 and CONSORT 2024 reporting guidelines …

Individualized therapy trials: navigating patient care, research goals and ethics

PB Kane, M Bittlinger, J Kimmelman - Nature medicine, 2021 - nature.com
Abstract 'Individualized therapy'trials (sometimes called n-of-1 trials) use patients as their
own controls to evaluate treatments. Here we divide such trials into three categories: multi …

Nutrigenomics research for personalized nutrition and medicine

J Kaput - Current opinion in biotechnology, 2008 - Elsevier
Current nutritional and genetic epidemiological methods yield 'risk factors' on the basis of
population studies. Risk factors, however, are statistical estimates of the percentage …

Enhancing quality and impact of early phase dose-finding clinical trial protocols: SPIRIT Dose-finding Extension (SPIRIT-DEFINE) guidance

C Yap, J Rekowski, M Ursino, O Solovyeva, D Patel… - bmj, 2023 - bmj.com
SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) 2013 provides
guidance for clinical trial protocol writing. However, neither the original guidance nor its …

Within-person variation in serum thyrotropin concentrations: main sources, potential underlying biological mechanisms, and clinical implications

E van der Spoel, F Roelfsema… - Frontiers in …, 2021 - frontiersin.org
Background Individuals exhibit fluctuations in the concentration of serum thyroid-stimulating
hormone (TSH) over time. The scale of these variations ranges from minutes to hours, and …

A priori justification for effect measures in single-case experimental designs

R Manolov, M Moeyaert, JE Fingerhut - Perspectives on Behavior Science, 2022 - Springer
Due to the complex nature of single-case experimental design data, numerous effect
measures are available to quantify and evaluate the effectiveness of an intervention. An …

Guidelines for cellular and molecular pathology content in clinical trial protocols: the SPIRIT-Path extension

TJ Kendall, M Robinson, DJ Brierley, SJ Lim… - The Lancet …, 2021 - thelancet.com
Summary The 2013 SPIRIT (Standard Protocol Items: Recommendations for Interventional
Trials) Statement provides evidence-based recommendations for the minimum content to be …

Personalized (N-of-1) clinical trials for inflammatory bowel disease: opportunities and challenges

S Honap, G Zou, S Danese, L Peyrin-Biroulet… - Clinical …, 2024 - Elsevier
Abstract Background and Aims Heterogeneity of treatment effects in inflammatory bowel
disease means that many individuals or patient subgroups depart from the average for …