E-Rare will organize a workshop on data sharing and harmonization in order to promote those issues in the rare disease research community. The workshop will take place on the 4th of May in Berlin, Germany at the Ramada hotel and will be preceded by a poster session in the late afternoon of the 3rd of May.

For more information.

You can register until the 7 of April 2017 and submit an abstract for a poster HERE.

 

Orphanet (www.orpha.net) is a unique resource, gathering and improving knowledge on rare diseases so as to improve the diagnosis, care and treatment of patients with rare diseases.

To mark its 20th anniversary, the Orphanet website is undergoing a complete makeover, starting with the look and feel of the site.

 

E-Rare associates with Blackswan Foundation to co-organize 4th International Congress on Reaserch of Rare and Orphan Diseases - RE(ACT) 2018.

The congress will take place from 7-10 of March 2018 in Bologna, Italy.

 

European Medicines Agency has published the final guidance document for applicants to Horizon 2020 topic “New therapies for rare diseases” on the EMA website. Find more information HERE

Results of Joint Transnational Call 2016

Results of Joint Transnational Call 2016

In December 2015 E-rare launched the eigth E-Rare joint call (JTC 2016) for funding multilateral research projects on rare diseases on “Clinical research for new therapeutic uses of already existing molecules (repurposing) in rare diseases”. Fifteen countries joined this call: Austria, Belgium, Canada (including Québec), France, Germany, Hungary, Israel, Italy, Latvia, Poland, Portugal, Romania, Spain, Switzerland, and Turkey.

The aim of the call was to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach.

The process included a two-step submission and evaluation procedure.

In the first step a total of 30 eligible proposals were submitted. After careful examination by the Scientific Evaluation Committee (SEC), 20 proposals were selected for the second stage of evaluation. Each of the full proposals have been evaluated by at least two SEC members and two additional external experts during the first stage of evaluation and the reviews were sent to project coordinators in order to give them the opportunity of studying the assessments and commenting on experts' arguments and evaluations for the second stage of evaluation. Each of the full proposals and the answer to the reviews have been evaluated by at least three SEC members for the second stage of evaluation.

Following the second SEC evaluation and ranking of the best projects, 8 consortia with a foreseen budget of about 11.2 Mio € were selected for funding.

Selected projects:
DPem
Title: Dimethylfumarate for the treatment of bullous pemphigoid
Coordinator: Ralf Ludwig

HCQ4Surfdefect
Title: Hydroxychloroquine (HCQ) in pediatric ILD
Coordinator: Matthias Griese

NICOFA
Title: Nicotinamide for the treatment of Friedreich ataxia
Coordinator: Jörg Schulz

REALS
Title: Repurposed Enoxacin for the treatment of patients with Amyotrophic Lateral Sclerosis
Coordinator: Eran Hornstein

ReDox
Title: Repurposing doxycycline in the treatment of AL amyloidosis
Coordinator: Giovanni Palladini

ROCK-ALS
Title: Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS
Coordinator: Paul Lingor

ROPROP
Title: Propranolol for preemptive treatment of threshold retinopathy of prematurity
Coordinator: Christoph Bührer

TAMDMD
Title: Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial
Coordinator: Dirk Fischer

E-Rare 2012 - Created by Toussaint Biger