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In celebration of the worldwide Rare Disease Day 2023 (RDD23), the global multistakeholder policy initiative Partnering4PNH is delighted to invite you to the event “Addressing the unmet medical needs of people living with a rare disease: What will it take?”. During...
Defined as affecting less than 1 in 2,000 people, rare diseases impact the lives of over 30 million people in Europe. Earlier this year, the European Commission proposed a Revision of the 20-year old legislation on medicines for children and rare...