The Global Cell & Gene Therapy Summit
Cell and gene therapy represent intersecting fields of biomedical research with the shared goal of treating, preventing, or curing diseases. These approaches are aimed at understanding the underlying causes of […]
Racial and Ethnic Diversity in Pediatric Clinical Trials: Current Barriers and a Path Towards Inclusive Clinical Trial Design and Conduct
Webinar objectives: 1. Understand the role that historical and ongoing marginalization play in research participation 2. Discuss how research findings in homogeneous populations are not generalizable to all children 3. […]
CMT Clinical Trial Readiness Summit
As new clinical trials emerge for Charcot-Marie-Tooth disease (CMT), the importance of optimizing measures, endpoints and the participant experience has never been more urgent! Hereditary Neuropathy Foundation (HNF) is committed […]
Clinical trials in rare disease: The role of patient registries
Therapies for rare diseases are emerging at a rapid pace yet rigorous evidence regarding their safety, efficacy, and effectiveness in practice is often sparse and is difficult to generate. High […]
European Conference on Rare Diseases 2024
The European Conference on Rare Diseases and Orphan Products (ECRD) is the largest, patient-led, rare disease policy-shaping event held in Europe. By bringing together people with rare diseases and patient […]
RARE Bootcamp
The RARE Bootcamp is designed for patients and advocates who have started funding rare disease research and are looking to better coordinate and build structure around their efforts. For more […]