Drugmakers, patient advocate groups, policymakers and others have come together for Rare Disease Day, which took place at the end of February, to both celebrate progress and call for more action in this therapy area.
In the UK, Genetic Alliance sought to highlight the fact that rare diseases are collectively common, although individually rare, with data showing that 1 in 17 people will be affected by a rare condition at some point in their lives.
The group is currently working on research projects including an evaluation of rapid genome sequencing (rGS) for seriously ill children at Great Ormond Street Hospital NHS Foundation Trust and the Institute of Child Health.
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