Rare disease research is difficult due to the scarcity of patient information on the conditions. Because there are so few people affected, patient recruitment for clinical studies might be very difficult. To address these difficulties, a number of charities, patient advocacy organisations, and government agencies have developed online registries to collect patient data for researchers, doctors, and public health experts to use. It's crucial to remember that study into the aetiology, processes, and treatment approaches of rare diseases can help to advance advancements in common diseases. Rare diseases are biologically complex, and scientists are working hard to figure out what causes them and how they progress clinically. As researchers identify the molecular and genetic drivers of many diseases, we've witnessed amazing progress in the creation of medicines to treat patients with rare diseases.
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