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.

Programs to access to clinical compounds

Programs to access to clinical compounds

In order to access to clinical compounds, pharmaceutical companies like AstraZeneca or Pfizer, are developping programs to propose number of their molecules that could be used in repurposing studies.

AstraZeneca Open innovation Program

In addition to marketed and Ph 3 compounds listed on the company’s website, AstraZeneca invites meritorious scientific proposals to study selected additional Ph 2a ready compounds (mechanisms) listed on their Open Innovation platform in a Clinical Table of Compounds.  To discuss the suitability of one of these compounds and, if mutually deemed warranted, obtain a ‘Letter of Support’ for AstraZeneca to supply the formulated compound as well as matched placebo contact Dr. Craig Wegner at craig.wegner[at]  For clarity, review for and funding of the final full proposal would still be through this E-Rare-3 Call for Proposals, but AstraZeneca would, assuming they agree, provide compound, technical expertise related to the compound as well as clinical study, and supportive regulatory documents.

More information

Pfizer Compound transfer program
Pfizer has an extensive pure substance library that encompasses all of their publicly known compounds. You can submit a request for a Pfizer compound by going on INSPIIRE portal. Each request regarding use of their compounds will be reviewed for content, value, and consistency with Pfizer's direction for the requested compound. For approved requests Pfizer executes Compound Transfer Agreements (CTAs) that provide the compound free of charge to a third party to conduct the agreed upon study. Processing time from initial submission to the approved compound(s) shipment to your facility may take up to 12 weeks.

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E-Rare 2012 - Created by Toussaint Biger