Highly purified cannabidiol for epilepsy treatment: A systematic review of epileptic conditions beyond dravet syndrome and Lennox–Gastaut syndrome

S Lattanzi, E Trinka, P Striano, C Rocchi, S Salvemini… - CNS drugs, 2021 - Springer
Background Cannabidiol (CBD), which is one major constituent of the Cannabis sativa plant,
has anti-seizure properties and does not produce euphoric or intrusive side effects. A plant …

Redefining the hypotheses driving Parkinson's diseases research

SL Farrow, AA Cooper, JM O'Sullivan - NPJ Parkinson's disease, 2022 - nature.com
Parkinson's disease (PD) research has largely focused on the disease as a single entity
centred on the development of neuronal pathology within the central nervous system …

4-Aminopyridine is a promising treatment option for patients with gain-of-function KCNA2-encephalopathy

UBS Hedrich, S Lauxmann, M Wolff… - Science translational …, 2021 - science.org
Developmental and epileptic encephalopathies are devastating disorders characterized by
epilepsy, intellectual disability, and other neuropsychiatric symptoms, for which available …

A call to arms against ultra-rare diseases

ST Crooke - Nature Biotechnology, 2021 - nature.com
A call to arms against ultra-rare diseases | Nature Biotechnology Skip to main content Thank
you for visiting nature.com. You are using a browser version with limited support for CSS. To …

[HTML][HTML] Establishment of an international collaborative network for N-of-1 trials and single-case designs

J Nikles, P Onghena, JWS Vlaeyen, RK Wicksell… - Contemporary clinical …, 2021 - Elsevier
In this article we briefly examine the unique features of Single-Case Designs (SCDs)(studies
in a single participant), their history and current trends, and real-world clinical applications …

Drug development for psychotropic, cognitive-enhancing, and disease-modifying treatments for Alzheimer's disease

J Cummings - The Journal of neuropsychiatry and clinical …, 2021 - Am Neuropsych Assoc
Alzheimer's disease (AD) is a progressive neurodegenerative disorder with limited available
therapies. There is progress in developing treatments for neuropsychiatric indications in AD …

From diagnosis to treatment in genetic epilepsies: Implementation of precision medicine in real-world clinical practice

M De Wachter, AS Schoonjans, S Weckhuysen… - European Journal of …, 2024 - Elsevier
The implementation of whole exome sequencing (WES) has had a major impact on the
diagnostic yield of genetic testing in individuals with epilepsy. The identification of a genetic …

Meeting the needs of patients with ultrarare diseases

ST Crooke - Trends in Molecular Medicine, 2022 - cell.com
Patients with ultrarare diseases present unique challenges to the health care systems of
developed economies that demand novel approaches, beginning with achieving a …

[HTML][HTML] Toward responsible clinical n-of-1 strategies for rare diseases

VM Defelippe, GJMW van Thiel, WM Otte… - Drug Discovery …, 2023 - Elsevier
N-of-1 strategies can provide high-quality evidence of treatment efficacy at the individual
level and optimize evidence-based selection of off-label treatments for patients with rare …

Study protocol: combined N-of-1 trials to assess open-label placebo treatment for antidepressant discontinuation symptoms [FAB-study]

A Müller, S Konigorski, C Meißner, T Fadai, CV Warren… - BMC psychiatry, 2023 - Springer
Background Antidepressant discontinuation is associated with a broad range of adverse
effects. Debilitating discontinuation symptoms can impede the discontinuation process and …