The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
October 25th 2024
On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.
Elucidating the Role of Type I Collagen Mutations on Respiratory Function in Osteogenesis Imperfecta
September 17th 2024Better known as brittle bone disease, osteogenesis imperfecta can cause severe respiratory distress at birth and longterm effect to respiratory function. Morello shares his team's research into the cause of this effect.