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 : 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