One drug to treat many diseases: unlocking the economic trap of rare diseases.

Journal Information

Full Title: Metab Brain Dis

Abbreviation: Metab Brain Dis

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Metabolism

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

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"Conflict of interest The authors declare no conflicts of interest."

Evidence found in paper:

"Funding The work of the authors on rare diseases is supported by the National Science Center (project grant nos. 2017/25/N/NZ2/00812 to KP and 2017/25/B/NZ2/00414 to GW). KP acknowledges the award from the L’Oréal-UNESCO Program “For Women in Science”, and the scholarship from the Foundation for Polish Science within the program “Start”. The funding sources had no involvement in this work at any stage and in the decision to submit the paper for publication. Neither the medical writer nor the editor has been involved in the preparation of this paper."

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Tool: rtransparent

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Last Updated: Aug 05, 2025