We are excited to be participating in the World Orphan Drug Congress US and are looking forward to meeting you there and discuss the challenges and opportunities to bring rare disease therapies to patients faster.
Join us on July 12 for our sessions:
11:55 AM EDT: Treatment and Regulatory Challenges of Systemic Juvenile Idiopathic Arthritis – What Can We Do Better?
Perspectives from patient/parent advocacy, regulatory consultation and clinical trial operations
- Systemic juvenile idiopathic arthritis as a rare and occasionally lethal disease
- Clinical background and patient stories
- Systemic JIA Foundation – new generation of patient/parent advocacy and research support group
- Haemopoietic stem cell transplantation as an ultimate cure – what do we know and what we do not know?
- Treatments and regulatory challenges addressing unmet needs
- Clinical trial operational challenges – new technologies, decentralized trials
- How can clinical research service company help?
2 PM EDT: Roundtable: Patient Focused Drug Development: Meeting unmet needs by combining research with practice
- Maria-Cruz Morillo, Global Therapeutic Operations Lead, Rare Diseases, Allucent
- Chris Watson, Director, Consulting, THREAD
- Richard Vesely, Vice President, Regulatory Strategy, Allucent
- Luciana Peixoto, Member of Parents Advisory Board, Systemic JIA Foundation