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| http://dx.doi.org/10.1503/jpn.240010 | DOI Listing |
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[Statistical design and application of clinical trials with small sample sizes for rare diseases].
Zhonghua Liu Xing Bing Xue Za Zhi
December 2024
Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing211166, China.
Due to the limited number of cases, conducting large-scale clinical trials for rare diseases is challenging. This review introduces several small sample statistical designs tailored for rare diseases, including crossover design, -of-1 design, randomized placebo-phase design, randomized withdrawal design, group sequential design, and adaptive design. It discusses the advantages, disadvantages, and application scenarios of these designs.
View Article and Find Full Text PDFJoining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics.
Ther Adv Rare Dis
September 2024
Dutch Center for RNA Therapeutics, Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Antisense oligonucleotides (ASOs) offer versatile tools to modify the processing and expression levels of gene transcripts. As such, they have a high therapeutic potential for rare genetic diseases, where applicability of each ASO ranges from thousands of patients worldwide to single individuals based on the prevalence of the causative pathogenic variant. It was shown that development of individualized ASOs was feasible within an academic setting, starting with Milasen for the treatment of a patient with CLN7 Batten's disease in the USA.
View Article and Find Full Text PDF"It Attracts Your Eyes and Brain": Refining Visualizations for Shared Decision-Making with Heart Failure Patients.
Appl Clin Inform
October 2024
Department of Medicine, Weill Cornell Medicine, New York, New York, United States.
Article Synopsis
- N-of-1 trials provide a personalized way for patients to compare the effectiveness of treatments using their own health data, particularly in the context of managing heart failure with preserved ejection fraction (HFpEF).
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Making use of N-of-1 trials to treat ADHD in people with psychosis: a hypothetical case.
J Psychiatry Neurosci
April 2024
From the Douglas Mental Health University Institute, Department of Psychiatry, McGill University, Montréal, Que., Canada (Dalton, Joober, Karama, Palaniyappan); and Robarts Research Institute and Department of Medical Biophysics, Western University, London, Ont., Canada (Palaniyappan).
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