Clinical Trials Often Exclude Patients with Rare Diseases, Highlighting a Critical Gap
Families grappling with rare diseases often find that clinical trials, designed to test new therapies, are frequently inaccessible to them. This reality is underscored by the experience of families like mine, as I navigate the complexities of my granddaughter’s condition, Dravet syndrome, a severe form of epilepsy linked to genetic mutations. Dravet syndrome, primarily caused…









