The Oxalosis & Hyperoxaluria Foundation (OHF) and Dicerna Pharmaceuticals invite you to participate in a webinar on Tuesday, August 15, 2017 at 16.00 hours GMT to discuss research presented by Dicerna during the 12th International Workshop on Primary Hyperoxaluria in Tenerife, Spain in July.
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
Ask a question by emailing: email@example.com (subject line: Dicerna Webinar) by Sunday, August 13, 2017.
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 National Renal Complement Therapeutics Centre in Newcastle has launched a new website – www.atypicalHUS.co.uk – containing information for both patients and clinicians on atypical haemolytic uraemic syndrome and C3 glomerulopathy. The site contains information about the diagnosis and management of aHUS, including patients who have end-stage renal failure and may benefit from the prophylactic use of eculizumab at time of renal transplantation.
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.
IgA Nephropathy: driving innovation with a rare renal disease registry.
A National Cystinosis Study Day will be held on Tuesday 19th September 2017 from 9.30am – 4.30pm at the Post Graduate Centre, Queen Elizabeth Hospital, Birmingham.
Patients and Health Professionals with an interest in Cystinosis are welcome.
To register for a place, please contact Michelle Lowe – Michelle.firstname.lastname@example.org
Cystinosis Patient Day 2017
The RaDaR Operational Management Board and Rare Disease Group Leads have agreed to apply for an Ethics amendment to clarify the fact that RaDaR consent covers Patient View sign-up, subject to availability at individual renal units.
The proposed amendment will also allow for data linkage to other registries, including the Hospital Episode Statistics (HES) and Office for National Statistics (ONS) databases. This will allow for a far more comprehensive RaDaR data-set than is currently available with manual data entry. It will also allow for longitudinal follow-up of patients who have previously consented to condition-specific research studies, or who do so in the future.
In addition, it is proposed to ask patients to consent to being contacted directly by a member of the central RaDaR team, regarding information events or further research studies. Such communication would always be approved by the patient’s Consultant before being sent out and will largely be circulated via Patient View.
Approval will be sought from NHS Digital’s data Access Request Service (DARS).
It is aimed to submit the Ethics amendment by the of September. Please continue to recruit patients to RaDaR in the meantime, using the current consent documents.
Further details will follow in the coming weeks but we would welcome suggestions over the proposed amendment to Melanie.Dillon@RenalRegistry.nhs.uk.
RaDaR features as a Case Study on the new Understanding Patient Information website, with the focus on supporting research into rare kidney disease.
The latest issue of the NephroS Newsletter has details on the current accrual and a report from the recent Patient Information day in Bristol back in March.
NephroS Newsletter June 2017
The PKD Charity are organising an ARPKD Family Day on Saturday, 22nd July 2017 at Uffculme Centre, Queensbridge Road, Moseley, Birmingham B13 8QY from 10 am – 3pm.
The event will include:
- Keynote talks about ARPKD from the experts
- Roundtable discussions between parents, patients, doctors & scientists
- Supervised entertainment & activities for children and young people at the Marvellous Superstar Summer Party organised by Birmingham Children’s Hospital
Travel bursaries are available.
ARPKD Family Info Day 2017 Flyer