Pharmaceuticals used for the diagnosis, prevention, and treatment of rare diseases are known as orphan drugs. Rare diseases differ from common diseases in that they have a low prevalence rate and so appeal to a small patient population. These medications are being developed to treat people with extremely rare disorders for which there is now no therapy option. Pharmaceutical inventors are often discouraged from developing treatments for extremely limited patient groups due to the increasing expense of medication research in tandem with rigorous regulations, as well as the low return on investment. Orphan diseases are rare diseases that affect small patient groups and are consequently "orphaned" by the pharmaceutical industry, with only a few authorized pharmacological therapy choices available. Incentives for orphan drugs have sparked research into diseases with a high unmet medical need.
Title : The foundation for rare disease and its role in the european rare disease research landscape
Daniel Scherman, Foundation for Rare Diseases, France
Title : The effect of Eupalinolide B on amyotrophic lateral sclerosis: A case report
Wang Huaixiu, Shanxi Provincial Hospital, China
Title : Progress related in genetic research on kawasaki disease
Jiao Fuyong, Shaanxi Provincial People’s Hospital, China
Title : Covid-19 seems to be Initiated by the heparan-sulfate dysregulation by coronavirus: The use of low-molecular- weight heparin (LMWH) can prevent and treat covid-19 when it Is used in early stages, as a heparan-sulfate-regulating medicine
Fereshteh Sedaghat, Sedaghat Memory Clinic, Iran (Islamic Republic of)
Title : Lumevoq gene therapy in leber hereditary optic neuropathy
Magali Taiel, GenSight Biologics, France
Title : Drug recommendation system using a collaborative filtering in machine learning
J. Somasekar, Jain University, India