The RealiseD project launched a new multi-stakeholder survey that invites the rare disease community to share insights and help improve clinical trial enrolment
The course will focus on the current status and advancements of clinical research especially to address unmet medical needs. Innovative methodologies, regulatory challenges, and the involvement of adolescents in clinical trials will be discussed.
A new simulation framework leverages pharmacometrics to optimise clinical trial designs for disease-modifying treatments in rare neurological conditions.
A continuous–categorical Item Response Theory framework integrates clinical and digital outcomes to improve disease severity estimation in degenerative ataxias, increasing sensitivity and reducing sample sizes in rare disease trials.
Join us for an insightful webinar where we tackle one of the biggest hurdles in rare disease research: generating evidence that truly informs decisions.
