Rare Rights. Common Cause.
Hosted by Cambridge BioMarketing in partnership with Akcea Therapeutics and Boston Public Health Commission and Rare Disease United Foundation
In the U.S., there are over 7,000 diseases classified by the FDA as “rare.” In the U.S., these diseases affect nearly 20 million people, more than HIV and cancer combined. While their conditions are diverse, all rare patients and their families face the serious pathology of their diseases while surviving in systems—medical, governmental, societal— that are not currently designed to address their needs. Despite these challenges, patients and families are fueling research to drive the discovery of new therapies and to provide access to existing therapies with life-changing benefits. Not only are they fighting for their lives, they’re fighting for their rights—as people, as patients, and as citizens.
During this panel, Cambridge BioMarketing Director of Medical Strategy, Dr. Samuel Falsetti, will moderate three experts representing the community, medical science, and biotechnology to discuss how all healthcare stakeholders can take the rare-disease-as-a-right challenge into their own hands and play a role in advocating for improved recognition and care. They will highlight the importance of communication amongst patients, advocacy groups, healthcare providers, insurers, government and industry to improve rare disease education and access to appropriate healthcare.