Future of Rare Diseases Research
It is still a reality for rare disease patients to spend years in the health care system before an accurate diagnosis is made. Next-generation sequencing and improved data sharing have resulted in faster discovery of gene disease and thus diagnostics. However the rate of gene disease discovery is now slowing probably as a consequence of the likely complex origin of the remaining unsolved disease. To face this challenge, new methods in particular addressing non coding region of the genome will need to be developed. In addition, even if the rate of rare disease therapeutic development and approval has been increasing, 94% of rare diseases still need an approved treatment. In addition, the number of untreatable uncommon diseases that have a first treatment approved remains low and even when a treatment is available, patients do not have always a ensured access to it. To face therapeutic discovery challenges, new approaches such as data-mining and repurposing and new models for funding drug discovery and covering treatment costs will be needed.
- Patient access to diagnosis
- International network for undiagnosed patients
- Potential advancements in therapeutic development
- Rare disease mechanism discovery
Related Conference of Future of Rare Diseases Research
February 27-28, 2019
2nd Global Experts Meeting on Infectious Diseases
| Radisson Hotel Narita | Tokyo, Japan
March 13-14, 2019
12th World Congress on Virology and Infectious Diseases
| Holiday Inn Atrium | Singapore
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April 08-09, 2019
2nd Global experts meet on STD-AIDS and Infectious Diseases
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April 10-11, 2019
3rd International Conference on Influenza and Emerging Infectious Diseases
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9th Asia Pacific STD and Infectious Diseases Congress
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Future of Rare Diseases Research Conference Speakers
Recommended Sessions
- Clinical Research on Rare Diseases
- Living with a Rare Diseases
- Rare Disease
- Rare Pediatric Diseases
- Treatment for Rare Diseases
- Challenges in Rare Diseases
- Future of Rare Diseases Research
- Infectious Diseases
- Mystery Diagnosis of Rare Diseases
- Neglected Tropical Diseases
- Neurology Rare Diseases
- Orphan Drug
- Plant Pathology
- Rare Cancer Diseases
- Rare Diseases in Aging
- Rare Diseases in Brain
- Rare Diseases in Endocrine System
- Rare Diseases in Eye and Ear
- Rare Gastrointestinal Diseases
- Rare Haematology Diseases
- Rare Immune System Diseases
- Rare Oral Diseases
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