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April 2014

The latest news from DEBRA International

April 2014

Calling for the EB community to work together on EB clinical practice guidelines

Two doctors, standing back to back, and holding folders

Over the last few years DEBRA International has been shaping a project to develop clinical practice guidelines (CPG) for the major aspects of EB medical care, e.g. wound management, oral health care, pain management etc. (For more on what exactly a clinical practice guideline is, see our article entitled 5 things you need to know about clinical practice guidelines). We recently put out a call to clinical experts and DEBRA organisations to get involved and help us progress this valuable work. The response was excellent and thanks to those of you who got in touch.

We still need help however, in the form of clinical experts to take on the task of leading guidelines, additional clinical experts to play supporting roles, patients to participate and assess the guidelines for usefulness and DEBRA organisations to provide funding. For more on what is involved and how to get in touch, please see this overview on the DEBRA International website

A guiding handbook for people living with EB

Screenshot of the homepage of the EB Handbook

In another great initiative from our colleagues in Austria, Dr. Anja Diem, Dr. Katharina Ude-Schoder and Mag. Brigitte Sailer, BA (all of the EB House in Salzburg) have developed an online EB Handbook.

This handbook has been created to help people directly affected by EB deal with the many challenges that the condition brings. It provides an overview of the different types of EB, along with advice on a variety of topics, including managing pain, caring for newborns and eye care. It also provides access to a beautifully written chapter on everyday life with EB, which tackles a range of topics, including clothing, playing sports and vacations and discusses issues which might arise at each of the major life stages.

Read more

A focus on DEBRA Iran: No EB patient should be left alone

Screenshot of DEBRA Iran's website

Dorothee Rahn, journalist for DEBRA International, interviews Dr. Amir Barzgar, on establishing the EB House in Iran.

"We should all be closer together" - what a wonderful sentence to close an interview and to start a very special article. This week, I was lucky enough to speak to Amir, who lives nearly 3.000 miles away from where I was doing the interview. Thanks to the internet and modern technology, it was possible to speak to him as if he was in the next room.

Amir is a paediatric dermatologist, working with about 350 registered EB patients in Iran. But getting to the point of being able to support EB patients and their families in that part of the world was not always easy: "We have a lot of bureaucracy." Getting a new charitable organisation officially registered requires a lot of paperwork and takes a lot of time getting it approved by the Iranian government. Read more

United for the cause: Brazilian EB groups grow together as a DEBRA

Group picture of delegates from 4 Brazilian EB patient groups

In October 2013, the first Southbrazilian EB Symposium took place in Blumenau, Brazil. It brought together more than 90 people from four patient support groups working for people with EB.

They have now concluded to continue as one united DEBRA Brazil, who is just about to become a member of DEBRA International.

Read more

DEBRA supporting research: an update

Jury meeting in a conference room
Meeting of DEBRA International's Medical and Scientific Advisory Panel (MSAP)

In the first of two calls this year for EB research proposals, an exceptionally high 18 applications have been received from researchers around the world. External experts will be asked to review the proposals over the next 10 weeks and DEBRA's Medical Scientific Advisory Panel (MSAP) will meet to consider the proposals in June. The panel will assess all applications for scientific excellence, feasibility and the potential for patient benefit and will make recommendations to DEBRA on which proposals to fund.

In parallel, we are delighted to inform you that planning for DEBRA International's next triennial research conference is underway. It will be held May 3 – 6 2015 in Atlanta, Georgia, and will be co-organised by DEBRA International and DEBRA of America. Read more

Promising cream for butterfly children (EBS) receives "orphan designation" status

Yellow tube of Diacerein cream

There is no cure at present for Epidermolysis bullosa (EB) but healing and alleviating approaches are being intensively researched world-wide. Current treatment is limited to the alleviating of symptoms, careful dressing of wounds and relieving pain. Yet here as well, new, even more effective options are needed.

The research team of the EB House Austria has now conducted laboratory tests on the active substance, Diacerein, a molecule of the rhubarb root, for its effect on EB simplex (EBS). The promising results were then confirmed in a clinical pilot study with EBS-affected patients and published. Read more

The limelight on research for Rare Disease Day

Research assistant Emma Batten giving information at a stand (The Royal Holloway University of London), which is decorated with DEBRA UK logo and posters
Information stand at The Royal Holloway University of London

Many DEBRA organisations around the world got involved in this year's Rare Disease Day, on 28th of February. In addition to the national events, DEBRA International Research Manager, Dr. Clare Robinson took part in some activities to highlight the importance of EB research.

Clare was invited to The Royal Holloway University of London to speak about EB, to biology students and school teachers. The focus of the talk was on the hope being created by regenerative medicine research, for people with EB. Along with other rare-disease charities, DEBRA also had a stand at the event, where DEBRA UK Research Assistant, Emma Batten, and nurse, Caroline Mackenzie, were on hand to speak to school students about EB research and nursing care for people with EB. Read more

Unproven cell therapy from the Stamina Foundation in Italy now being re-evaluated

Chalkboard with "Stamina?" written on it

Leading stem-cell experts have raised concerns about unproven stem-cell therapies for some time. In 2013, the market approval of one such therapy, created by the Italian medical charity Stamina Foundation, led to extensive debate. The market approval of this controversial procedure was first blocked by the Italian Medicine's Agency, AIFA, due to the above stated concerns. However, patients’ hopes were high, and some suspected the authorities of depriving them of something that could improve their quality of life or even be life-saving. Patients successfully lobbied the Italian government to allow the use of Stamina therapy in public hospitals. Read more

Five things you need to know about EB clinical practice guidelines

Collage of a photo of 2 doctors holding folders, and the covers of 3 clinical practice guidelines for EB

DEBRA International is very focused on the development of clinical practice guidelines for EB but what are they and how do they help? Here are five things you should know about them.

  1. Clinical practice guidelines do just as the name suggests - they guide clinicians (doctors, nurses, dentists, physiotherapists etc.) in their decision-making, as they care for people with EB.
  2. Each EB guideline will contain recommendations for the best path to choose, when faced with a particular clinical challenge. The ideal scenario is for these recommendations to be based on scientific evidence, i.e. where different possible actions have been tested scientifically. Failing that, the expert opinion of clinicians working in the field is drawn upon.
  3. Clinical practice guidelines are not just for clinicians. With a condition as rare as complex as EB, patients become the true experts in their own condition. Clinical practice guidelines therefore, are also for patients who wish to understand more about their own medical care.

Read more

A special Thank you

Two women in an office: DEBRA Germany staff Bettina Höflein and Susanne Köhl
DEBRA Germany staff: Bettina Höflein and Susanne Köhl

We would like to say a special thanks to the DEBRA Germany Board for recent generous support of the clinical guidelines project.

We would also like to thank DEBRA Switzerland for their generous donation to international EB research.

DEBRA International is the umbrella organisation for a worldwide network of national groups, working on behalf of those affected by the rare genetic skin blistering condition epidermolysis bullosa (EB).

DEBRA International Executive Committee 2015/2016
Cinzia Pilo (Italy) – President; Loreto Moore (Chile) – Deputy President; Shoaib Gopalani (Canada) – Treasurer; Brett Kopelan (USA) – Deputy Treasurer; Jimmy Fearon (Ireland) – Secretary; Anna Kemble-Welch – Deputy Secretary; Mia Werkentoft (Sweden); Michael Jaega (UK); Vassil Nikolov (Bulgaria)

Internal Auditors
Graham Marsden (UK); Rainer Riedl (Austria)

Michael Fitzpatrick (Australia) – CEO; Lea Prujean (New Zealand) – International Coordinator/ EB Without Borders; Clare Robinson (seconded by DEBRA Austria) – Head of Research; Avril Kennan (seconded by DEBRA Ireland) – Project Manager/ Clinical Practice Guidelines; Barbara Dissauer (seconded by DEBRA Austria) – Communications and Research Manager; Hayley Epps (seconded by DEBRA UK) – Communications Manager; John Dart (voluntary) – Senior Advisor to the Executive Committee

DEBRA International is a registered charity: ZVR 932762489

Contact: Am Heumarkt 27/1 | 1030 Vienna | Austria | Tel: +43 (0)1 876 40 30
e-mail: office@DEBRA-international.org | www.debra-international.org
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