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Given the broad scope and varying definitions of rare diseases worldwide, our analysis focused on the top ten rare diseases with the highest levels of research and development (R&D) activity 14

1 2 "GnRH agonists in the treatment of symptomatic endometriosis: a review"

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Although desnutrin/ATGL mRNA is downregulated in obese rats (130), adiponutrin mRNA is induced 50-fold (9)

However, long-term safety data is limited, and its full risk profile is not yet completely understood

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