On Monday 2nd September 2019 a joint patient and clinician event was held at in London to discuss current and future research around the RaDaR rare disease registry, as well as ways to boost patient engagement in research.
The agenda, a report from the day and pdfs of the presentations can be found below.
As well as taking part in the RaDaR Rare Disease Registry, we recommend that patients who are interested in research visit the NIHR’s new Be Part of Researchwebsite which allows you to search for studies by condition and/or location.
Studies that are being led or endorsed by Clinicians involved in RaDaR are advertised on the Patient Information pages for each condition.
Help us bring together millions of people across the world and unite with one powerful voice for kidney health awareness.
World Kidney Day in the UK is led by the Kidney Charities Together group (Kidney Care UK, Kidney Research UK, NKF (Kidney Patients UK) and the PKD Charity). Help us bring together millions of people across the world and unite with one powerful voice for kidney health awareness.
Over 180 support packs have already been sent out to renal and transplant units as well as individuals and patient groups supporting the day by organising an awareness activity or event.
What are you planning, following the 2019 global theme of Kidney Health for Everyone Everywhere?
At the meeting, Dicerna announced the expansion of its drug development programs to encompass all forms of primary hyperoxaluria (PH). Based on new pre-clinical data presented in Tenerife, the investigational therapy DCR-PHXC has the potential to treat all forms of PH.
The webinar will provide an overview of the following research topics:
• The need for new treatment strategies for all types of PH
• The role of the LDHA gene in the pathogenesis of PH
• The DCR-PHXC preclinical data demonstrate potent, durable, and precise knockdown of LDHA in animal models of PH1, PH2 and idiopathic PH, and show a simple, direct linear relationship between LDHA inhibition and oxalate production
• Clinical development plans and timelines for DCR-PHXC
• Kim Hollander, Executive Director, Oxalosis & Hyperoxaluria Foundation
• Dr Sally-Anne Hulton, M.D. Paediatric Nephrology, Birmingham Children’s Hospital UK
• Ralf Rosskamp, M.D. Chief Medical Officer, Dicerna Pharmaceuticals
The Summer issue of the RaDaR Newsletter features updates from the IgA Nephropathy and aHUS groups, details of a Cystinosis patient information event in Birmingham and important information about the forthcoming Ethics amendment.
The IgA Nephropathy Rare Disease Group have had an article published in this month’s issue of the Journal of Kidney Care. The article discusses how having access to an international rare disease registry with data on this disease can help improve kidney care around the world.
The IgAN research group in Leicester has been given £2.7 million to establish the world’s first international registry of IgA Nephropathy patients. The registry will follow the same format as RaDaR and will collect clinically characterised data from IgA Nephropathy patients across Europe, Asia and South America.