HEIDELBERG 12th January 2016 – First Meeting of the European Reference Network for Rare Renal Disease (EuRenNet)
Twenty participants in this first informal meeting of members of the future European reference network for rare kidney disease, from nine countries. The meeting was chaired by Pr. Franz Schaeffer Heidelberg, and was sponsored by the Working Group on Inherited Kidney Diseases of the ERA-EDTA, coordinated by Pr. Olivier Devuyst, Chairman of the FEDERG Scientific Council.

Patients are heavily involved in the preparatory work for future network. FEDERG and three representatives of national associations, federation members, were present at the meeting.

The objective of this meeting was to take stock of the application process, according to the latest instructions issued from the Commission, in particular the “Toolbox” put online on 23rd December 2015.

The meeting has highlighted the challenges that the teams will face for the national accreditation of their reference centers. Much work will have to be done by each center candidate to provide documentation for obtaining highest score on the evaluation criteria of reference centers (Health care providers), which are severe according to the professionals.

The project is then a bet on the future, since there are no European funds to support the network at this stage of the process and because those who have already done a great job for organizing their national reference networks are solicited more, with benefits in other countries.

Although this noble task of solidarity is perceived by everyone as heavy, the participants have formally pronounced for rapid work to enable the network to be ready when the call will be launched in a few weeks.

FEDERG presented the patient organizations perspectives: patient’s organisations are stakeholders for the rare renal ERN, and motivated for its development. They see it as an opportunity to expand access to good health care to all patients in Europe. This is a strong solidarity objective. The principle is that the expertise goes as close to the patient no matter the level of organization of the country in which the patient lives. FEDERG also sees the ERN as a process that will enhance organization of patients across Europe, and yield to greater mobilization for registries and trials with more patients.

In summary: “Better care for all and then better research”

View of the Participants at the meeting

IMG_6206 (1)

FEDERG representatives, de gauche à droite : Daniel Renault (Chair), Claudia Sproedt (Vice-Chair),
Marjolein Storm (NVN), Marjolein Bos (Cystinosis Netherlands & NVN).

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KDIGO Controversies Conference on Complement-Mediated Kidney Diseases
Barcelona, November 19-21, 2015

KDIGO guides are the best known among professionals (doctors and researchers) worldwide. The
organization KDIGO born in USA, invites doctors and researchers, and the most prestigious experts in
each renal disease.

At this conference, we ASHUA have been fortunate to participate with 2 people, and representing
FEDERG and all of European aHUS patients. Due our agenda, Mireya (Secretary of ASHUA) was all
time participating all time on the Treatment Strategies Group, while I was on the second day.

For two full days, doctors and researchers, discussed the questions, detailed below, made by the
other professionals involved in KDIGO worldwide. The findings of the whole debate, we shall know
once drafted the new guidelines for the management of aHUS.

As an organization and representatives of aHUS patients, we have known as health professionals,
their interest in improving the lives of patients, their dedication … but above all, their willingness to
work with us the patients.

We were able to chat and exchange points of views with many Doctors, worldwide, although in the
case of aHUS, perhaps research and good management of aHUS, are concentrated in Europe and

We highlight several conversations … with Dr. Goodship, which have made possible apart from our
attendance at this Conference, he paid much attention to our comments and concerns,
encouraging us to continue to participate in future KDIGO conferences, representing all kidney
diseases, but also sharing all KDIGO jobs with all patients, in all countries.

Due to technical and medical concepts spoken, I do not feel appropriate to summarize here two full
days of debate among professionals, we shall have it soon, when editing the new guide. Although
all comments between doctors and researchers may I highlight the following:

  • Dr. Ardisino (Italy). A case of cold in a patient with Eculizumab, DO NOT stop the medicine,
    perhaps even increase the dosage.
  • Dr. Ruggenenti (Italy). When a liver transplant is performed at an aHUS patient, Eculizumab is
    strongly recommended during and after transplant until the new liver produces all new
    proteins (especially factor H) in sufficient quantity to avoid new onset aHUS in the following
    days transplantation.
  • Dr. Loirat (France), Dr. Ariceta (Spain) and Dr. Appel (USA). When a debut of aHUS,
    immediately suspected it is aHUS, administer Eculizumab, not loose time, especially in
  • Dr. Noris (Italy). Genetic analyses help to understand the origin of the aHUS. May it is time to
    start to customized treatments depending of each aHUS patient, but due we don’t know
    much about that, we should take care and learn step by step. We must be careful with the
  • Dr. Blasco (Spain). Patients … associations are the key to request and pressure administrations
    to create reference hospitals and further enhance the investigation, which should be
    centralized and organized.
  • Dr. Van de Kar (Netherlands). She presented the new video made between aHUS patients
    and doctors (coordinated by the association NVN), to raise awareness throughout the
    population, but also to the administration and doctors. We have shown our Personal ASHUA
    bracelet, and she liked a lot and paid special interest in it as she sees many advantages for
    all patients.
  • Dr. Fadi Fakhouri (France). Eculizumab discontinue??? There are cases is completely
    discouraged as the factor H, but for other cases is highly recommended to continue
    treatment for at least one year, and from there continued again between 6 or 12 months
    once the renal function has stabilized. Once discontinued, the patient should be monitored
    very strictly.
  • Dr. Carla Nester (USA). PNH patients take many more years with Eculizumab, so in some cases,
    data can be compared with aHUS cases and learn.
  • Dr. Véronique Frémeaux-Bacchi (France). The visibility of patients is essential, she liked very
    much our ASHUA red bracelet and the campaign we are doing with celebrities.


Group 5. Treatment Strategies, breakout group questions.

  1. What is the optimal duration for eculizumab treatment in aHUS patients? What is the evidence
    that justifies life-long therapy especially in adults?
  2. What is the evidence for optimal dose and dose interval for eculizumab and are there
  3. What is the optimal treatment for patients with aHUS due to anti-FH antibodies?
  4. How do we stratify C3G patients for novel agents and clinical trials?
  5. What should the current approach to treatment of C3G be?
  6. Kidney transplantation in aHUS and C3G
    • What is the best time frame for kidney transplantation after the onset of end-stage
      renal disease (ESRD) in aHUS or C3G patients treated with anti- cellular therapy and/or
    • What is the risk of recurrence?
    • What is the best strategy in order to prevent aHUS (C3G) recurrence after kidney
      transplantation: preventive vs. pre-emptive strategies?
    • Should living kidney donors be used (preferentially?); living related?
  7. How should eculizumab treatment be monitored: CH50, AP50, free eculizumab or total
    eculizumab, in vitro EC tests?
  8. What is the optimal treatment for rapidly progressive or bad prognosis histology C3G?
  9. What is the role for immunosuppression in the treatment of C3G
  10. What is the role of eculizumab in the treatment of C3G? a. In what subgroups might it be


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ERN, the move is starting!

The European Reference Networks, ERN, the move is starting!

“ERN” an acronym you need to get familiar with!

EURORDIS organized in Paris on the 27-29th October, the annual meeting of the Council of European Federations (CEF), the meeting was very well attended this year because of the focus on the establishment of the European Reference Network for Rare diseases. Daniel Renault Chair of FEDERG participated to the meeting.

Recall that Eurordis has played a key role in the EUCERD committee, (the European Committee of Rare Diseases) that have paved the way to ERN.

The situations for the ERN pre-set themes are much diverse. There are thematic for which the patients organizations are scattered (for 10 themes of ERN there is, for each theme, between 20 and 100 associations members of EURORDIS) with a similar divided situation for the clinicians.

Fortunately the theme for which we are concerned “rare renal diseases” (RRD) is in a better situation on both sides. Only 10 renal associations in EURORDIS (out of 610), half of them are already member of FEDERG. Thus FEDERG is somehow legitimate to represent the patients in the ERN project on RRD.

EURORDIS will make efforts in the coming weeks to get all the groups organized into Patient Advocacy Group, that will be done for ERN but also looking beyond. Again Rare Renal is already almost done.
Officially the fist call is to be launched before March ends with 3 months for making the proposal. EURORDIS urges us to speed up the preparation steps.

For Rare Renal, a core team of nephrologists and geneticians together with FEDERG will start the preparatory works mid November. A partner meeting is to be held 12th January 2016 in Heidelberg at the invitation of Franz Schaeffer.

Photo 1 Yann Le Cam Director of EURORDIS presenting the ERN process and how patients should be organised.

Photo 2 The CEF audiences with Matt Johnson, Scientific Director of EURORDIS, presenting the process of submission and validation of the ERN projects.

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Alport Meeting in Göttingen

THE INTERNATIONAL WORKSHOP for ALPORT SYNDROME RESEARCH STRATEGY is ON:  Susie Gear from Alport UK and FEDERG and Professor Oliver Gross adressing the audience at the inaugural session Friday 25th 2015 Gottingen.

Some 100 participants, Nephrologists, Geneticists, patients’ representatives and pharmaceutical companies get together in Gottingen GERMANY at the invitation of Professor Oliver GROSS to shape the research for ALPORT SYNDROME.

During 3 days discussion will focus on Science and on how best organise the Alport Syndrome Community with the appropriate infrastructure for research (Registries, biobanks, etc..).

FEDERG is taking an important part in that endeavour through mainly Susie Gear from Alport UK, Daniel Renault from FEDERG and AIRG-France, Catherine Cabantous from AIRG-France.
The future of ALPORT Syndrome treatment will be discussed around the following 4 themes:
1   Basic and Translational Science, Gene/Chaperone Therapy
2    Diagnosis and Genetics
3     Standard of care, Repurposing Therapy, Carriers, Management of Extrarenal Symptoms
4     Future Therapy, Registries, Biobanks, Clinical Trial


Some impressions

IMG_6044 IMG_6042 IMG_6043 IMG_6030

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FEDERG present at the 48th Scientific Meeting of the European Society of Paediatric Nephrologists (ESPN) from the 3 to 5th September 2015 in Brussels.

The organizers offered a boot during the 3 days of the Congress. Thanks to AIRG-Belgium the logistic for FEDERG went smooth. The last day of the Congress FEDERG hold a Members Meeting in the premises of the Congress.

This has been an opportunity to make our presence known of the European nephrologists and of some pharmaceutical companies that were in the Hall of exposition.

The members meeting addresses several issues related to FEDERG mandate and management. The last meeting was held in June 2013, and this was an opportunity for the members to express how they see FEDERG in future. An important issue discussed has been the on-going initiative on the creation of European Reference Networks for Rare Renal Diseases.

On the scientific side of the congress more than 400 contributions, the Nephrotic syndrome was the focus of several sessions, this is a frequent paediatric disease. For the renal genetic diseases, a significant number of contributions on aHUS. Also noteworthy a joint session ESPN and ERA-EDTA (Adult) on APKD and the new possibilities for treatment. See full program at http://www.espn-2015.org/program.php

Photo on the booth (left to right): Michel Schenkel, Daniel Renault, Marjolein Bos, Tess Harris and Marjolein Storm.

Members meeting (left to right): Daniel Renault Michel Schenkel, Bruno Woitrin, Tess Harris, Marjolein Bos and Marjolein Storm.

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Invitation European Symposion on Eye Problems in Cystinosis, October 8 – 10, 2015

Cystinose-Selbsthilfe e.V. and Cystinosis foundation Germany (a new founded foundation to support our patient organization and the cystinosis medical center Traunstein) invite you for a symposion on eye problems in cystinosis patients taking place October 08 – 10, 2015 in Salzburg / Austria.

As you can see in the attached flyer, there will be international specialists meeting to see a lot of patients and discuss several problems treating eye problems in cystinosis. Nephrologists and eye specialists from many countries will be there to follow this and learn from each other. The goal is to find guidelines for treatment that will be used worldwide, so all patients have the chance to be treated as it is best for them from day 1.

Now we thought this could be interesting for patient organizations too. So we would like to invite you to follow this symposion, listen to the specialists and take part in discussions. To keep this in a comfortable size, we will be happy to welcome two persons from every patient group in Europe or from every European country if there is no organization. We hope you will inform the patients in your country about the outcome of the conference.

Depending on how many participants will be there we will try to organize a short sightseeing tour as well. Salzburg has a lot of nice places to see and is known as the birth town of Wolfgang A. Mozart.

Salzburg has an international airport and can be reached by train or car of course.

For further questions please don’t hesitate to ask me or Anja Karl at Kinderklinik Traunstein who will be happy to help you. Her phone and email address are given on the flyer.


Download Flyer


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ERA-EDTA, 52nd Congress

ERA-EDTA is organising its 52nd Congress in London May 28 -31 2015. Tess Harris from PKD International will also represent FEDERG at this event.

ERA-EDTA is a European professional organisation (more than 7000 members) focussing on the advancement of medical science and of clinical work in nephrology, dialysis, renal transplantation, hypertension and related subjects.

ERA-EDTA is concerned by renal genetic diseases in many ways. One of the working group of ERA-EDTA deals with inherited Kidney Disorders (WG-IKD) ; This group is chaired by Pr. Olivier Devuyst, who is chairing the Scienticfic Council of FEDERG.

Link to the WG on IKD http://era-edta.org/wgikd/ERA-EDTA_working_group_on_Inherited_kidney_disorders.htm

The link to the website of the association is http://www.era-edta.org

The scientific program can be downloaded on the following link


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FEDERG is on its way!

The launching meeting of the FEDERG’s initiative – Federation of European patient groups affected by Renal Genetic Diseases (RGD) – was held at the initiative of AIRG-France on May 26, 2012 as a dedicated meeting within the 49th ERA-EDTA Congress held in Paris, Palais des Congrès.

The launching meeting was supported by ERA-EDTA (European Renal Association and European Dialysis and Transplantation Association), by ESPN (European Society of Pediatric Nephrologists), by EURORDIS, and by AIRG-France.



The meeting brought together 10 nephrologists, some had made the trip especially for the meeting FEDERG, and 10 representatives of associations of renal genetic diseases.

The meeting has been an opportunity for all participants of the launching, to express and share unanimously the desire to see the emergence of a European federation. The willingness to join forces is present, and is ready to concretize itself and expand to others.

The first part of the meeting chaired by Professor Yves Pirson, Chairman of the Scientific AIRG Belgium, focused on the perspective of nephrologists about the importance of considering the genetic renal diseases in a European context .The speakers were Professor Dominique Chauveau, Chairman of the Scientific Committee, AIRG-France, Prof. Oliver Gross, Nephrologist Göttingen, also representing the German group of patients on Alport syndrome, Dr Elena Levchenko of Leuven, representing pediatric nephrologists and the society ESPN, and  Prof. Olivier Devuyst Zurich, Head of the Working Group of the ERA-EDTA on Inherited kidney disorders.

Each speaker was able to propose and develop strong arguments that advocate for an efficient organization at the European level of all patient groups and associations with a wide scope of diseases (renal and multi-organs) and not restricted to a single pathology.

The second part of the meeting, chaired by Daniel Renault, President AIRG France, was dedicated to identify the motivational elements of the associations represented and initialize the following preparations in the construction of the federation. Associations were represented either directly by the members present (some excused because of health problem) or by nephrologists attending the conference: – 8 countries of the EU-27 were represented: Germany – Belgium – Bulgaria – Spain-France – Ireland – Netherlands – United Kingdom; – 2 outside countries: Switzerland and Morocco as well as two international federations.

In the short time of two hours, the meeting allowed everyone to know each other and to outline areas of work and mobilization for the future. The collaboration between nephrologists and patient groups is recognized by everyone as the key to high efficiency. At the same time, it is recognized that it is hard to get patients mobilized in some contexts; therefore external support might be required. This should be one of the roles of FEDERG to support the emergence of local associations in national contexts unfavorable!

Beyond targeting a good collaboration between nephrologists and patient groups, the improvement of the “quality of life” for those who suffer from genetic kidney disease, has been emphasized by many as a priority. This should also be one of the focuses of a European federation.

Full reports of the meeting by clicking our AIRG-France website (see link below) and on the FEDERG web platform where you can directly contribute to and monitor the progresses of the federation.


One of the main recommendations of the meeting is to quickly enlarge the basis of FEDERG to more associations, covering more pathologies as well as more European countries.


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Our federation reached another milestone on 4th April 2014: the act is signed.

Photo 1:  Lawyer Mr. Watillon, Manuella Delplanck (AIRG-Belgium), Michel Schenkel (AIRG-Belgium), Secretary FEDERG.

Photo 2:  Bruno Woitrin (AIRG-Belgium), Treasury FEDERG, Michel Schenkel (AIRG-Belgium), Secretary FEDERG.

As this initial stage, we have 12 constitutive associations and one associated member, which is by far a real success.

The next steps are:
– The registration of the act and of the proxies. When done, we will have copies of the act.
Submitting the documents for the royal decree to the Minister. When  accepted it will be submitted to the king’s signature.


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