International Conference on Orphan Drugs and Rare Diseases
Revolutionary Changes
Regrettably, "rare disease" has historically been low on the public health agenda, a fact that the 350 million individuals worldwide who suffer from these diseases are well aware of. This is unsurprising since, while each of these rare diseases has too little a health burden to make a simple health-economic justification, rare diseases are thought to be just as common as other disorders. Rare disease detection and management is an example of an area that has profited from technological advancements. The integration of new medicines for rare diseases into existing care systems, on the other hand, calls into question a translational medicine premise that there is a linear pathway from bench to bedside. In awareness of the impact of patterns of clinical interventions and associated health services on patients' and their families' quality of life, a range of patient-oriented outcomes may be most relevant to examine for incremental therapies.
- Recent Trends
- Novel Innovations
Sergey Suchkov
The Russian University of Medicine & Russian Academy of Natural Sciences, Russian Federation
Vicki Ratner
ESSIC - International Society for the Study of BPS, United States
Kondakova O. B
National Medical Research Center for Children's Health Federal state autonomous institution of the Russian Federation Ministry of Health, Russian Federation
Paul L Kaufman
University of Wisconsin-Madison, United States
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