North American edition of the RE(ACT) Congress 2019 to be held at the Sheraton Center Toronto Hotel in Toronto, Canada, from May 8-11 2019. This 5th edition, the first to be held in North America will include sessions on clinical trials and registries, generalizable rare disease therapeutic approaches, gene editing, cell & gene therapy, stem cells, genetically modified cell therapies/clinical applications of gene therapies indigenous populations and rare diseases, and patient-focused drug development. REGISTER NOW! 

The European Joint Programme on Rare Diseases (EJP RD), which is the successor of E-Rare launches the Joint Transnational Call 2019. The call will  open officially in December. This year projects should focus on accelerated diagnosis and/or exploration of disease progression and mechanisms of rare diseases. More info under

28 February 2019 will be the twelfth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organisations from countries and regions all over the world will hold awareness-raising activities. The theme for Rare Disease Day 2019 is 'Bridging health and social care'.

For most people living with a rare disease, as well as their family members or carers, the reality of daily life can include any combination of the following: collecting and taking medicines, attending appointments, participating in physical therapy, using specialist equipment and accessing various social and community support services and respite care. Managing these care-related tasks alongside their usual daily activities such as work, school and leisure time can be challenging.

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

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

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

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

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

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

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

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

E-Rare 2012 - Created by Toussaint Biger