Study to Understand Fatal Genetic Diseases in Children Incorporates Digital Participant Education Program
A clinical trial was launched last week to help researchers understand how two rare and fatal genetic diseases develop in children and provide insight into treatment for them in the future. The study, run by Azafaros, will follow patients in the UK, US, France, Italy, Germany and Brazil for four years.
The PROspective Neurological Disease TrajectOry study (PRONTO) is a Natural History Study that follows children who have been diagnosed with GM1 or GM2 gangliosidoses (also known as Tay-Sachs and Sandhoff disease), a class of rare inherited disorders that are fatal in childhood and which currently have no approved treatments.
Read more about this new clinical trial here.
AI in Clinical Trials: A Long, But Promising Road Ahead
May 29th 2024Stephen Pyke, chief clinical data and digital officer, Parexel, discusses how AI can be used in clinical trials to streamline operational processes, the importance of collaboration and data sharing in advancing the use of technology, and more.
Including Women of Childbearing Age in Clinical Research
March 26th 2024In recognition of International Women's Month, we're featuring this recent talk between Associate Editor Miranda Schmalfuhs and Marie Teil, Global Head of UCB’s Women of Childbearing Age Program. They speak about the specific challenges women with chronic illnesses face when accessing appropriate treatment and participating in clinical trials, UCB's Women of Childbearing Age Program and it’s most successful strategies, and much more.
