Alisa Shakarian of Cambridge Biomarketing discusses the challenges pharma and biotech companies face in bringing drugs into the rare disease market.
Sam Falsetti, PhD of Cambridge Biomarketing discusses some of the biotech and rare disease successes of 2017, including the emergence of RNA therapies with the approval of Exondys 51 for Duchenne muscular dystrophy, as well as gene and CAR-T therapies that have had significant FDA approvals in 2017.
Alisa Shakarian of Cambridge Biomarketing discusses the role of Advocates in rare diseases and the challenges of diagnosing rare diseases.
Sam Falsetti, PhD of Cambridge Biomarketing discusses the film Rare in Common, which follows the experiences of rare families: their struggles, their strength, and their hope for the future as they confront the challenges of living with a rare disease. The movie has shown at several film festivals.