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.

Consultation on guidelines on Good Clinical Practice for Advanced Therapy Medicinal Products

Consultation on guidelines on Good Clinical Practice for Advanced Therapy Medicinal Products

The European Commission is launching an online targeted public consultation on the draft guidelines on good clinical practice for Advanced Therapy Medicinal Products (ATMPs) addressed particularly to small and medium-sized enterprises (SMEs), academia, hospitals and patient organisations.

The Commission has to draw up guidelines on good clinical practice specific to advanced therapy medicinal products. The document will focus on ATMP specificities only and will apply to the GCP Guidelines of The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).

The comments are invited to be sent by 31 October 2018. Responses should preferably be sent to sante-pharmaceuticals-B5(at)

Find more information here.

E-Rare 2012 - Created by Toussaint Biger