United Kingdom : PSR Orphan Experts, Ergomeds rare disease specialist division, organises fundraiser to support Global Rare Disease Day 2018.
As part of its commitment to the rare disease patient community, PSR Orphan Experts will be hosting a beach volleyball tournament to raise money and awareness for children suffering from epidermolysis bullosa (EB), a partly hereditary and incurable skin condition. Themed Sport mee voor het vlinderkind (Lets play sport for the butterfly child), the event will take place at Aalsmeer beach on March 6, 2018. The funds raised will go to Debra Nederland, a Dutch patient organisation for people with EB. As the slightest friction of simple day to day activities for example, showering, chewing, cuddling or wearing clothes or shoes may cause blisters on the skin of EB patients, children with this disease are called butterfly children their skin is as tender as butterfly wings. In Europe, 30,000 patients are estimated to be affected by this disease.
Despite general acknowledgement of the benefits brought by innovative therapies, patients suffering from rare diseases continue to face difficulties in receiving a correct diagnosis and in getting timely access to adequate treatment and care, said Roger Legtenberg, Chief Executive Officer of PSR Orphan Experts. We are proud to be able to support Debra Nederlands initiative with this years fundraiser as they represent the interests of people with EB and aim to achieve the best possible quality of life and care for the patient and their family.
Despite the constant growth in orphan drug development over the past 10 years most of the 7,000 rare diseases, many of which are genetic, and the majority of patients with rare diseases remain underserved. Because of the rarity, heterogeneity and the complex patient management associated with rare conditions, creating and executing an orphan drug development programme presents some of the highest hurdles in medicine. In recent years, there has been a rapidly growing demand for trials in rare and orphan indications because of legislation in both the US and the EU that has established incentives to support research in these areas, making it more financially viable, and the growth of both precision and preventative medicine.
Andrew Mackie, Chief Business Officer of Ergomed, said: Rare disease clinical trials are not just smaller versions of conventional clinical trials. Orphan drugs require specialist development skills due to the small number of patients available, severity of the disease and often acute nature of symptoms. Ergomed and PSR Orphan Experts, therefore, joined forces to create a leading global orphan drug development expert team that will allow drug developers to implement patient driven solutions in their clinical trials.
On Rare Disease Day, PSR Orphan Experts usually supports a local rare disease organisation, that could benefit from better awareness and funding. PSR Orphan Experts hopes to be able to expand this annual initiative for rare diseases throughout other regions of Europe and MENA. In addition to this annual event, PSR Orphan Experts is also active at other local events and last year coordinated the Rare Disease Symposium in The Netherlands which focused on the prospects for rare disease patients in 2030 in order to improve the future optimisation of orphan disease treatments.
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