Many rare diseases manifest themselves in childhood, resulting in a shortened lifespan and leading to a dependency on care throughout the patients’ lives, causing significant suffering to the patients and their families. Patients affected by rare diseases often spend years enduring a “diagnostic odyssey” before receiving the correct diagnosis, if ever. Most rare diseases lack effective treatments representing an enormous unmet medical need. EU research and innovation is crucial to accelerate the discovery and development of novel therapeutics. Restrictions: ©The material may not be edited and must be shown entirely.

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