Primary Hyperoxaluria Webinar, 15th August

 

 

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

Speakers

• 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

Participate

Questions

Ask a question by emailing: julieb@ohf.org (subject line: Dicerna Webinar) by Sunday, August 13, 2017.

New aHUS website

 

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.

Cystinosis Study Day, Tuesday 19th September

 

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 LoweMichelle.lowe@uhb.nhs.uk

Cystinosis Patient Day 2017

Proposed Ethics Amendment for RaDaR

 

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.

ADPKD Info Day, 1st July, London

 

Place: Monckton Lecture Theatre, St George’s Hospital, Cranmer Terrace, Tooting, London SW17 0RE
Time: Saturday 1st July 2017, 9:30 am to 4 pm
Hosted by: Dr Joyce Popoola, Dr Anand Saggar and the PKD Charity

  • Learn about ADPKD (Autosomal Dominant PKD) and the latest research
  • Find out more about the new drug, Jinarc (tolvaptan)
  • Put your questions to the experts
  • Meet and share your experiences with others
  • Everyone welcome – patients, families and carers

Lunch and refreshments included. There is no charge, but a donation is welcome to help cover the costs.

Book online at Eventbrite.

ARPKD Family Day, 22nd July, Birmingham

 

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