Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) is an ultrarare genetic condition causing developmental epileptic encephalopathy characterized by seizures and motor and intellectual disabilities. No disease-modifying therapies are available, and treatments focus mainly on symptom management to improve quality of life. The aim of this study was to better understand the burden of CDD based on family caregivers’ perceptions.

Digital health solutions (DHS) are technologies with the potential to improve patient outcomes as well as change the way care is delivered. The value of DHS for people with diabetes is not well understood, nor is it clear how to quantify this value. We aimed to summarize current literature on the use of patient-reported outcome measures (PROMs) in diabetes as well as in selected guidelines for Health Technology Assessment (HTA) of DHS to highlight gaps, needs, and opportunities for the use of PROMs to evaluate DHS.

People with Parkinson’s disease (PD) may use cannabis-based products for symptom management. In France, products containing tetrahydrocannabinol (THC) are prohibited, while cannabidiol (CBD)-products are readily available. However, data on cannabinoid use in French people with PD are lacking. To identify correlates of the use of cannabis-based products and to document their patterns of use and perceived effects.

In our global survey, we sought to expand this understanding to people living with LC in countries across different regions, and to bring a more comprehensive understanding of the care pathway experiences in terms of disease management and impact on their daily lives, treatment decisionmaking, stakeholder involvement, and gaps in information/support services. In light of COVID-19 and the mobilization of patients to engage more online, we also sought to leverage online patient communities as participants for our survey. This enabled access to additional “real-world” patient populations beyond traditional channels such as local recruitment agencies and patient organizations.