To guide public policy, rare diseases, a rising global public health concern, require an evidence-based estimate of global point prevalence. Forecasting disease outbreaks is extremely useful for efficiently planning and supplying resources. Rare diseases (RDs) are diverse, heterogeneous, and spread over the globe. Few are preventive or curable, the majority are chronic, and many lead to premature death. Despite their diversity, RDs have some characteristics in common due to their rarity, which needs a comprehensive public health strategy. The diversity of the data, which is derived from a variety of disparate information sources that are not standardized or difficult to combine, such as published case reports or systematic reviews, patient registries, expert opinions, and other anecdotal evidence, makes estimating the global point prevalence of RDs difficult. As RDs become a global policy objective of leaving no one behind, refinement of the epidemiological estimate of RDs is timely.
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