A rare disease, sometimes known as an orphan disease, is one that affects a small proportion of the world's population. Even if symptoms may not develop immediately, the majority of these rare diseases are inherited and present throughout a patient's lifetime. There has been an increase in the occurrence of rare diseases all across the world. An orphan drug is a pharmaceutical product used for the diagnosis, prevention, and treatment of various rare conditions or diseases, according to the European Organization for Rare Diseases. These diseases are distinct from other diseases in that they have a very low prevalence rate relative to other diseases, and so are purchased by a very limited patient population. The global orphan drug market was valued at US$ 147.56 billion in 2019 and is predicted to grow at a CAGR of 10% to US$ 413.36 billion by 2030.
Title : What does a technology-enabled patient concierge mean to the orphan drugs industry?
Harsha Rajasimha, Jeeva Informatics Solutions Inc, United States
Title : Improving regulatory confidence for formal early phase clinical development.
Candida Fratazzi, BBCR Consulting - Massachusetts, United States
Title :
Victor Shengkan jin, Rutgers University, United States
Title : Scleritis and systemic diseases: What should know the internist/rheumatologist ?
Philip Bielefeld, Avignon Hospital, France
Title : Progress related in genetic research on Kawasaki disease
Jiao Fuyong, Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Title : Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy
Magali Taiel, GenSight Biologics, France