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.

PARADIGM survey - share your opinion on patient engagement!

PARADIGM survey - share your opinion on patient engagement!


PARADIGM, a collaborative IMI project that aims to make meaningful patient engagement in medicines development a reality, such as through better tools and recommendations was launch last March. 


The objective is to develop processes and tools for three key decision-making points: research priority setting, design of clinical trials and early dialogue with regulators and HTA bodies.


To reach outcomes that are truly useful, a survey on patient engagement is being deployed and you/ your respective relevant colleagues have been identified as the target audience, specifically involved in the three areas of relevance for this project. 


PARADIGM would like to ask you to take a few minutes to share your opinion and experience on patient engagement.


Better patient engagement in medicines development means better health outcomes for everyone and we can’t afford to continue to take a fragmented approach to deliver fragmented results. We will reach this shared goal much faster if we are focused, build consensus and work together starting with the most important priorities.


Your answers will directly help in understand your needs, preferences and expectations. Share your opinion now!


The survey can be accessed HERE

E-Rare 2012 - Created by Toussaint Biger