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The European Commission is organising a 2nd conference on European Reference Networks on 8-9 October 2015, which will be hosted by the Ministry of Health in Lisbon, Portugal. Included as an event in the Luxemburgish Council Presidency programme, this conference will bring together highly specialised healthcare providers, experts, national authorities, decision–makers, and independent bodies with experience in the assessment and evaluation of healthcare providers.

The conference will focus on the implementation of European Reference Networks and facilitate the exchange of information and expertise on establishing and evaluating such Networks. It will look into the next steps of the deployment process, in preparation for the forthcoming call for European Reference Networks in 2016. This event builds on the success of the 1st conference on European Reference Networkswhich took place in Brussels on 23 June 2014.

More information on the conference, including the conference agenda, registration and webstreaming can be found here.

For more information on cross-border healthcare and ERNs:
http://ec.europa.eu/health/ern/policy/index_en.htm
http://ec.europa.eu/health/ern/docs/2014leaflet_ern_en.pdf

The Brains for Brain Foundation (B4B), the organisation leading the Inherited NeuroMetabolic Diseases Information Network (InNerMeD-I-Network) project, has recently participated at the workshop “Realising European Reference Networks for Rare Diseases: a preparatory workshop for the rare disease field”. The workshop organised by EUCERD Joint Action and the European Commission was held on 1-2 July 2015 in Bruxelles.

The workshop aimed to support the rare diseases (RD) field in preparing for the first Call for the implementation of European Reference Networks (ERNs), which is anticipated to be launched in December 2015.

The specific objective was to inform, assess and maximise the current state of ‘preparedness’ for RD ERNs.

ERNs are described in Article 12 of Directive 2011/24/EU (Directive on the application of patients’ rights in cross-border healthcare). The Directive acknowledges the unique potential of ERNs for the RD field by asking the European Commission to “support the development of European Reference Networks between healthcare providers and Centres of Expertise in Member States, in particular in the area of rare diseases” (Art.12). In addition, the Directive dedicates a separate Article to RD (Art.13).

The EUCERD Joint Action (EJA) is a 3-year-initiative coordinated by Prof. Kate Bushby and dedicated to supporting the  Commission Expert Group on Rare Diseases in formulating and implementing policies relating to RD. Since 2012, the EJA has organised workshops and prepared Recommendations and policy documents to support the RD field in maximising the opportunity afforded by ERNs. An EJA workshop in October 2014 focused on engaging Competent National Authorities and Member State representatives of the Expert Group on Rare Diseases.

Bringing together individuals with experience of coordinating/leading networks dedicated to specific diseases/disease areas requiring highly specialised expertise to deliver optimum clinical care to patients across Europe, the workshop ensured that clinical/academic representatives of the various disease groups have an opportunity to:

a)      Receive an update on the state of the art for RD ERNs, through presentations and insights from a range of experts, including the European Commission, the Tender contractors (elaborating the Manual and Assessment toolkit for ERNs, and also defining services for ERNs), and RD experts engaged with the ERN topic over the last four years.

b)      Express the needs and expectations of their particular disease community relevant to RD ERNs.

An interactive and dynamic discussion on crucial issues took place addressing focal questions such as:

• What is the current state of preparedness for RD ERNs, amongst the clinical and research communities of Europe?
• What will be the added-value of RD ERNs – what benefit will they bring for the stakeholders involved?
• What are the frequently asked questions surrounding ERNs and how might these be addressed for the RD field?
• How will the clinical communities integrate with other domains (e.g. specialised procedures, genetic laboratories, etc.)
• Given that the EC Expert Group on RD is recommending a model for grouping RD in the future ERNs, how do we decide what group certain diseases fall into? And how do we ensure cross-talk across all ERNs?
• How might ERNs interact with existing and future RD tools and resources, including registries, JRC European Platform on RD registration, RD Connect etc.?
• What should be the services of an ERN? What good practices exist in the current networks/disease fields Are there robust telemedicine systems in use for sharing RD data, images, scans etc.?
• What are the next steps?

During the workshop the state of the art of networking practices across the various RD or low prevalence and complex clinical ‘areas’ were shared and explored, with an emphasis on ensuring the best possible applications from the RD field result from the December 2015 Call for ERNs.

The second Official ERN Conference and associated workshops will take place in Lisbon this October (8-9th).

For more detailed info see:

Final Agenda for RD ERN Workshop 1-2 July 2015

Report ERN Workshop_1&2 July 2015

It is with great pleasure that we announce the Opening Ceremony of the New Center for Rare Diseases in Wiesbaden.

The B4B Foundation has contributed to create this Center. In fact,together with Christina Lampe, formerly working at Villa Metabolica, Maurizio Scarpa, President of the B4B Foundation, has created a Rare Disease Institute in Wiesbaden which has been opened on last January 1^st 2014.

The Centre takes care mainly of LSDs and neurometabolic diseases,  and is a teaching and a treating Unit for Neurometabolic Diseases.

Seminars and Awareness activities have been activated thanks to specific grants from Biomarin, SHIRE, Genzyme and Actelion.

After one year building phase, we will finally celebrate the activity of this important new Center for Rare Diseases at HSK, Dr. Horst Schmidt Kliniken in Wiesbaden.

The official ceremony  will take place on

 Wednesday, February 4th, 2015, 2 p.m.

 at Dr. Peter Jäger- Bildungszentrum 

(HSK, Ludwig-Erhard-Straße 100, 65199 Wiesbaden) 

Click on the following link  for more detailed information: 

Invitation-Center for Rare Diseases_EN_email

Souvenir of the ZSE Wiesbaden Opening Ceremony (1)

Official Opening Cerimony REPORT

On February 5-7th, 2015, we will have the 9th B4B Workshop in Frankfurt.  The meeting aims to discuss on:

• Research achievements in the field of neurodegenerative disorders at clinical and basic science level in the field of neurodegenerative lysosomal storage disorders and Blood Brain Barrier;
•  New recent advances on natural history and pathophysiology of LSDs particular attention to the important role of an early intervention in preventing the morbidity and mortality associated with each of the disorders;
• Factors which control the entry into the brain of medicines and other therapeutic agents which may be helpful in treating central nervous disease;
• How B4B might collaborate with the European Union to stimulate interest in the research on LSDs and BBB. For this reasons representatives from EU Commission will be invited;
• Collaborations with international family associations and corporations to increase knowledge about storage diseases and research projects;
• The role of the industries in driving innovation for new therapeutical approaches for true unmet needs.

 In particular a special session dedicated to B4B and InNerMeD European Action is scheduled for Saturday 7th February. This moment represents a great opportunity for presenting detailed information and updating about InNerMeD-I project recent achievements and discussing the importance of actively participating to InNerMeD-I Information Network. An enlarged Partners collaboration and the establishment of a joint effort to tackle iNMDs, is in fact essential to ensure that patients are given the priority they deserve and that their needs are met.

Please stay tuned for more updated info.

 9th B4B Workshop Scientific Programme

We are pleased to announce the  17th Barrier and  Transporter – Meeting taking place in Bad Herrenalb close to Karlsruhe, Germany, from  May 11^th to May 13^th 2015.

Subject of this workshop are membrane transport proteins in barrier organs, drug transport and drug delivery across biological membranes as well as screening models such as MCDK or Caco-2 cells.

This workshop might also be of special interest for PhD students to get in contact with participating colleagues from pharma industry.

Please visit the website https://sites.google.com/site/transportertage/home, where you can register until April 29^th and submit an abstract for poster or oral presentation.

The registration fee is 275,- € including 2 nights with complete room and board.

In case of question please call +49-6221-548336 or send an email to transportertage@googlemail.com

We are pleased to announce the  Lysosomal Diseases Gordon Research Conference that will be held in March 15-20, 2015
c/o  the historic Hotel Galvez – Galveston, Texsas.

This meeting will  mark the 60th anniversary of the development of the lysosomal disease concept by H.G. Hers in 1965. Emphasis will be placed on the burgeoning role of the greater lysosomal system as a key regulator of cell homeostasis in health and disease. Invited speakers will focus on newly emerging aspects of lysosomal biology, molecular and cellular mechanisms of pathogenesis and the relationship of lysosomal diseases to other disorders, the consequences of heterozygosity and of XCI in X-linked lysosomal disorders, new experimental therapies and experiences with ongoing clinical trials. There will also be a late-breaking topics session (with speakers chosen closer to the time of the meeting so as to include the most recent developments) and short poster talks (based on those posters generating the greatest interest during the meeting). In addition to invited speakers, each session will also have short talks with presenters chosen prior to the meeting based on abstract submissions.

APPLICATIONS DEADLINE

PRELIMINARY PROGRAM

For more info please visit  http://www.grc.org/programs.aspx?id=14946

We are pleased to announce the fourth Cold Spring Harbor conference on Blood Brain Barrier which will begin at 7:30 pm on Wednesday, December 10 and run through lunch on Saturday, December 13, 2014.

The purpose of this Cold Spring Harbor meeting is to explore and encourage new conceptual approaches and to apply novel methods to the neuroprotective physiologies of BBB structures. Our goal is pair genetic model organism biological methods with recent insights in vertebrate BBB physiology and development. We hope to create cross fertilization between fields that rarely see each other and get people thinking about how model systems can help parse the regulatory networks necessary for parsimonious BBB function.

The conference is intended to foster cross-disciplinary exchange of ideas and expertise between developmental and evolutionary biologists, vascular and BBB physiologists and disease oriented industry scientists interested in modifying or circumventing specific pathways of neuroprotection. While a number of speakers have been invited, please note that the majority of oral presentations will be drawn from openly submitted abstracts.

Organizers

Britta Engelhardt, University of Bern, Switzerland

Stefan Liebner, Goethe University Clinic, Germany
Ryan Watts, Genentech, Inc.

Keynote Speakers

Richard Daneman, University of California, San Francisco
Maiken Nedergaard, University of Rochester Medical Center

Topics    

• BBB Development
• The Functional BBB
• BBB in a Dish 
• Molecular BBB Transport
• The Diseased BBB
• Pericytes, Astrocytes and the BBB 

Discussion Leaders

Joan Abbott, Kings College London, UK
Dritan Agalliu, University of California at Irvine
Bruce Appel, University of Colorado
Robert Bell, Pfizer, Inc.
Elisabetta Dejana, Fondazione IFOM, Italy
Per-Ola Freskgard, F. Hoffmann-LaRoche Ltd, Switzerland
Robyn Klein, Washington University School of Medicine
Jeremy Nathans, Johns Hopkins School of Medicine
Eric Shusta, University of Wisconsin
Benoit Vanhollebeke, Universite Libre de Bruxelles, Belgium
Jack Wong, Houston Methodist Research Institute
Joy Yu, Genentech

For more info please visit  http://meetings.cshl.edu/meetings/2014/bbb14.shtml