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: firstname.lastname@example.org (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.
Due to unforeseen circumstances the National Cystinosis Study Day has had to be postponed. The event will hopefully be re-scheduled for Spring 2018. Details will be published here once they are confirmed.
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.email@example.com
Cystinosis Patient Day 2017
Prof Detlef Bockenhauer, chair of the Rare Disease Committee of the Renal Association, is hosting a forum to help clinicians and researchers to better understand RaDaR and Fabry Disease in particular. The event will be held during UK Kidney Week on Monday 19th June 2017 at the Pullman Hotel, Kings Dock, Liverpool, L3 4FP
The meeting has been organised by Sanofi Genzyme and will include a buffet dinner.
Places are limited and attendees are asked to RSVP to John Applegate at firstname.lastname@example.org
A Cystinosis Patient and Clinician Education Day will be held on Tuesday 19th September from 9.30 – 16.30 at the Postgraduate Centre, Queen Elizabeth Hospital, Birmingham.
Further details will be available nearer the time but to express an interest in taking part as a speaker please contact Michelle.Lowe@uhb.nhs.uk
RD-Connect, NeurOmics and EURenOmics are hosting a rare disease outreach day on 3rd May in Berlin, Germany. The event aims to brimg together stakeholders from the rare disease community to share the value of the tools created and the knowledge generated through these projects.
Invitation to Outreach Day