BioDigest: Biotechnology and Rare Diseases
Every day, organizations like the National Organization for Rare Disorders (NORD) work with patients and families to connect them with the resources they need to manage rare diseases and disorders. These resources range from support group recommendations to contacts at companies creating therapies for rare and orphan diseases and disorders. In this video, we meet a family with two children affected by rare disease, and learn how, with the help of NORD and a drug therapy created by BioMarin, they refuse to let their disease hold them back.