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Evaluation of chenodeoxycholic acid treatment in adult patients with cerebrotendinous xanthomatosis: A randomized, placebo-controlled Phase 3 study (RESTORE)
Abstract   Peer reviewed

Evaluation of chenodeoxycholic acid treatment in adult patients with cerebrotendinous xanthomatosis: A randomized, placebo-controlled Phase 3 study (RESTORE)

Andrea DeBarber, Yaz Kisanuki, Paulo Nobrega, Ryan Himes, Suman Jayadev, John Bernat, Vikram Prakash, James Gibson, Austin Larson, Paulo Sgobbi, …
Molecular genetics and metabolism, Vol.141(4), 108216
04/2024
DOI: 10.1016/j.ymgme.2024.108216

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