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One patient told me, "Dr

doi: 10.1038/s41586-021-03539-7 136 ZhongFLiangSZhongZ

DOI: Received: Accepted: Published: Publisher Name: Springer, Cham Print ISBN: 978-3-030-51519-5 Online ISBN: 978-3-030-51519-5 eBook Packages: Living Reference Biomedicine and Life SciencesReference Module Biomedical and Life Sciences

The findings aim to provide actionable insights for policymakers, clinicians, researchers and patients to advance DHT-driven, patient-centric approaches, ultimately improving patient outcomes and addressing the unmet medical needs of rare disease populations worldwide, ensuring they have equitable access to innovative treatments and improved healthcare