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Translational Science of Rare Diseases

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Welcome to the website of Translational Science of Rare Diseases, an international peer-reviewed journal publishing original research, timely reviews, conference summaries, and relevant commentaries on prospective therapeutics for patients affected by rare diseases. With an emphasis on translational medicine, the journal’s mission is to address the challenges of rare diseases and advance the development of innovative therapies to improve patient outcomes and quality of life.

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