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 www.ejprarediseases.org

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.

E-Rare Newsletter November 2015

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ERA-Net for Research Programmes on Rare Diseases
E-Rare Newsletter November 2015

The eighth E-Rare joint call for funding multilateral research projects on rare diseases (JTC2016) will be open on the 7th of December, 2015. The following 14 countries intend to participate in this call: Austria, Canada (including Quebec), France, Germany, Hungary, Israel, Italy, Latvia, Poland, Portugal, Romania, Spain, Switzerland, Turkey.

The specific objective of this call is to promote the clinical and pre-clinical proof of concept for the potential application of medicinal products in rare indications either already marketed or having achieved a significant stage in the development process. Two types of projects will be eligible for this call:
•    Type A: Preclinical studies to verify target engagement and to perform additional toxicity testing if necessary (for example in the case of paediatric indications where juvenile animal studies might be warranted) in a disease model for a maximum period of one year followed by the implementation of Phase 1b or Phase 2a clinical trials at the beginning of the second year of the project.
•    Type B: Milestone-driven Phase 2 clinical trials to demonstrate that the Agent modulates the target and has the potential to yield the desired clinical outcome in the proposed disease population for a period up to three years.
Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10.000 persons in the European Community, EC associated states and Canada.

The use of existing European health research infrastructures or initiatives is strongly encouraged when appropriate. The following European Research Infrastructures or Initiatives were identified as potentially useful for this kind of study: BBMRI; EATRIS; ECRIN; ELIXIR; EU-OPENSCREEN; INFRAFRONTIER; RD-Connect and European Medicines Agency. More information on European Research Infrastructures/Initiatives, their services and costs can be found on www.erare.eu/infrastructures/

ARE EXCLUDED: (1) Development of new models of diseases; (2) Discovery and early development of new compounds; (3) Feasibility studies of devices; (4) Preliminary studies of radiation, surgical, behavioural or rehabilitation therapies; (5) Studies on advanced therapies (gene therapy, cell therapy); (6) Clinical studies concerning rare infectious diseases, rare cancers and rare adverse drug events or secondary outcomes in treatments of common diseases; (7) Compassionate use.

The deadline for proposal pre-registration is February 1, 2016.
The deadline for proposal submission is March 3, 2016.

Visit our website www.e-rare.eu

Please Note:

The information provided in this pre-announcement is indicative, may be subject to changes and is not legally binding to the funding organisations.

Additional funding organisations might join the call before the official publication.

Interested applicants are encouraged to initiate scientific contacts with potential project consortium partners for applications.

In order to access to clinical compounds, pharma companies like AstraZeneca or Pfizer, are developing programs to propose number of their molecules that could be used in repurposing studies.
For more information on AstraZeneca Open Innovation Program and Pfizer Compound Transfer Program: Programs to access to clinical compounds

The 3rd international congress on research of rare and orphan diseases to be held at the Crowne Plaza Barcelona – Fira Center in Barcelona, Spain, from 9th to 12th March 2016 is jointly organized by E-Rare and the Blackswan Foundation. The congress will bring together world leaders and young scientists from stem cell, cell biology, gene therapy, human genetic, or therapeutic applications to present state-of-the-art research, to discuss results and to exchange ideas.
For more information: RE(ACT) congress 2016

On the 9th of March, before the official start of the RE(ACT) congress, E-Rare will organize a workshop dedicated to Interactions between EMA and RD researchers on pre-licensing activities in collaboration with the European Medicines Agency.
For more information: EMA workshop

www.e-rare.eu contact@erare.eu

E-Rare 2012 - Created by Toussaint Biger