Karen Peluso, NFNE Executive Director and NF patient Megan Duff will be speakers at the Rare Disease Day Program on Monday, February 23rd at the Massachusetts State House.
Karen will participate in the “Taking Stock of Rare Disease Research Today & Tomorrow” panel discussion. The panel will feature key leaders in orphan drug development, emphasize the importance of research and development in rare disease, and highlight the role that patient and advocates play in bringing much-needed therapies to market.
Megan will tell her story as a patient living with Neurofibromatosis type 1 along with two other rare disease patient advocates during the Rare Disease Day commemoration between 11:00 AM and 12:00 PM in the Great Hall.
The event is free and open to the public. Registration in advance is requested.
The program is as follows:
9:00 AM to 10:30 AM:
State of Rare: Taking Stock of Rare Disease Research Today & Tomorrow
- Ed Kaye, M.D., Senior Vice President, Chief Medical Officer, Sarepta Therapeutics
- Karen Peluso, Executive Director, Neurofibromatosis Northeast
- Steve Uden, M.D., Senior Vice President, Research, Alexion Pharmaceuticals
- Phil Vickers, Ph.D., Global Head of R&D, Shire
Moderator: Ken Dhimitri, Vice President, Operations, Boston Biomedical Associates
11:00 AM to 12:00 PM:
Join MassBio, VHL Alliance, member companies, patient groups, patients and legislators to recognize Rare Disease Day in the Great Hall.
- Chris Anselmo, Miyoshi Myopathy patient
- Megan Duff, Neurofibromatosis type 1 patient
- Lisa Cimino, mom of a Focal Segmental Glomerulosclerosis (FSGS) patient
- Senator Karen Spilka, Co-Chair of the Massachusetts Biotech Caucus
- Tim Boyd, National Organization of Rare Disorders
- Ilene Sussman, Executive Director, VHL Alliance (Emcee)