October 24, 2018
Article
Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.
October 18, 2018
RT001 has demonstrated arrest of disease progression in 2 patients with infantile neuroaxonal dystrophy.
October 17, 2018
Ellen Sigal, PhD, discusses how she helped make the concept of the expedited FDA development program a reality.
Karlyne Reilly, PhD, discusses the Rare Tumor Patient Engagement Network and the importance of getting patients with rare conditions involved in cancer research.