Government urged to approve life-changing drugs to treat rare diseases

Posted March 01, 2017

It said 80 per cent of the rare diseases have a genetic origin while 50 per cent affect children, most of whom do not live beyond five years. In Europe, a disease is defined as rare when it affects fewer than one in 2,000. That is why worldwide cooperation is so important. "ERNs have taken this joined-up approach a step further, allowing more formal and organised cooperation for the diagnosis and treatment of rare disease patients".

"From our perspective it is crucial that United Kingdom institutions can continue to work closely with their European counterparts so that United Kingdom and other European patients will not lose out", he argued. The center's team, a group of pharmaceutical and clinical research professionals with experience in the areas, will focus on "addressing the specific challenges presented by the small, widely dispersed populations of rare disease and pediatric patients", the release stated. If all the rare disease patients in the world came together, they would account for the third most populous country.

"Lack of awareness and scientific knowledge often leads to misdiagnosis or late diagnosis and delayed treatment". In 2015, 47 percent of novel new drug approvals were for rare diseases, and 566 medicines are in development for patients with rare diseases. "Clinical research will help us find better and more effective treatment for rare disease".

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The day is therefore an opportunity to call upon researchers, universities, students, companies, policy makers and clinicians to do more research and to make them aware of the importance of research for the rare disease community. ORDI recently held a public event Race for 7 to raise awareness for rare diseases.

"Rare Disease Day is an important day of recognition for those living with rare conditions".

About the National Organization for Rare Disorders (NORD)® The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases. Rare diseases and disorders are less likely to receive the funding and awareness required to accelerate research.