The NGO Committee for Rare Diseases is a UN’s global, multi-stakeholder initiative focusing on rare diseases. This is a major step to address the rare disease challenges on a global(…)
The 3rd conference of the International Rare Diseases Research Consortium (IRDiRC) will take place on February 8-9, 2017 in Paris,.
Elixir Excelerate addresses the infrastructure needs of the rare disease community. Take part in the survey to help prioritize the most important bioinformatics tools and better adapt ELIXIR services to(…)
All patient organisations are encouraged to join the European Patient Advocacy Groups to ensure that the patient voice is heard in the European Reference Networks.
We are pleased to announce the formation of a panel of experts who will consider the biobanks against a set of criteria to ensure the minimum quality assurance is met.
The last week of September in the UK is dedicated to rising awareness of Spinal Muscular Atrophy (SMA), an inherited neuromuscular condition.
The RD-Connect Guidelines for informed consent were acknowledged by IRDiRC as fundamentally important for the international rare diseases research.
In September 2016, the number of newsletter subscribers reached 1000! We would like to thank our readers for following our updates and being with us all that time.
Contribute to raising awareness and supporting Duchenne patients and families by donating a Duchenne Balloon today.
Identifying the cellular mechanism can be a breakthrough in the therapy for SMA. Employing genetic modifiers might revolutionise the treatment and give hope for better quality of life for the(…)