[HTML][HTML] Conduct and implementation of personalized trials in research and practice

RL Kravitz, N Duan - Harvard data science review, 2022 - ncbi.nlm.nih.gov
The mainstay of evidence development in medicine is the parallel-group randomized
controlled trial (RCT), which generates estimates of treatment efficacy or effectiveness for the …

[HTML][HTML] Personalized data science and personalized (N-of-1) trials: Promising paradigms for individualized health care

N Duan, D Norman, C Schmid, I Sim… - Harvard data science …, 2022 - ncbi.nlm.nih.gov
The term 'data science'usually refers to the process of extracting value from big data
obtained from a large group of individuals. An alternative rendition, which we call …

Individual Treatment Trials—Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?

AM Wiesinger, H Strobl, FB Lagler - Pharmaceuticals, 2023 - mdpi.com
Mucopolysaccharidoses (MPS) are a group of rare, heterogeneous, lysosomal storage
disorders. Patients show a broad spectrum of clinical features with a substantial unmet …

RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

AM Wiesinger, B Bigger, R Giugliani, C Lampe… - Pharmaceutics, 2023 - mdpi.com
Mucopolysaccharidosis (MPS) is a group of rare metabolic diseases associated with
reduced life expectancy and a substantial unmet medical need. Immunomodulatory drugs …

[PDF][PDF] An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis. Pharmaceutics 2023, 15, 1565

AM Wiesinger, B Bigger, R Giugliani, C Lampe… - 2023 - academia.edu
Mucopolysaccharidosis (MPS) is a group of rare metabolic diseases associated with
reduced life expectancy and a substantial unmet medical need. Immunomodulatory drugs …

[PDF][PDF] Investigation of N-of-1 trial models

P Bywood, J McMillan - 2021 - research.iscrr.com.au
Investigation of N-of-1 trial models Page 1 APRIL 2021 EVIDENCE REVIEW #280
Investigation of N-of-1 trial models Evidence review A review of the literature on the utility and …