Efficacy, safety, and tolerability of chenodeoxycholic acid (CDCA) in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized withdrawal, double-blind, placebo-controlled, crossover phase-3 study.

Kisanuki YY, Nobrega PR, Himes R, Jayadev S, Bernat JA, Prakash V, Gibson JB, Larson A, Sgobbi P, DeBarber AE, Murphy E, Fedor B, Wong Po Foo C, Dutta R, Imperiale M, Garner W, Quan J, Vig P, Duell PB, Perez S, Ramdhani RA, Saute JA, RESTORE Study Group Collaborators