1: Zhu Y, Lobato AG, Rebelo AP, Canic T, Ortiz-Vega N, Tao X, Syed S, Yanick C,
Saporta M, Shy M, Perfetti R, Shendelman S, Züchner S, Zhai RG. Sorbitol
reduction via govorestat ameliorates synaptic dysfunction and neurodegeneration
in sorbitol dehydrogenase deficiency. JCI Insight. 2023 May 22;8(10):e164954.
doi: 10.1172/jci.insight.164954. PMID: 37014713; PMCID: PMC10322690.
2: Perfetti R, Bailey E, Wang S, Mills R, Mohanlal R, Shendelman S. Safety,
Pharmacokinetics, and Pharmacodynamics of the New Aldose Reductase Inhibitor
Govorestat (AT-007) After a Single and Multiple Doses in Participants in a Phase
1/2 Study. J Clin Pharmacol. 2024 Nov;64(11):1397-1406. doi: 10.1002/jcph.2495.
Epub 2024 Jul 10. PMID: 38988185.
3: Bailey E, Phan H, Ahmad A, Thomas J, Ames EG, Pritchard AB, Quinonez SC, Wang
S, Dayley C, Salt A, Pick C, Durrant A, Johnson S, Nicodemus-Johnson J, Dickson
SP, Perfetti R, Hendrix SB, Shendelman S. Results of the ACTION-Galactosemia
Kids Study to Evaluate the Effects of Govorestat in Pediatric Patients with
Classic Galactosemia. J Clin Pharmacol. 2024 Nov 21. doi: 10.1002/jcph.6170.
Epub ahead of print. PMID: 39569553.
4: Randall JA, Sutter C, Wang S, Bailey E, Raither L, Perfetti R, Shendelman S,
Burbridge C. Qualitative interviews with adults with Classic Galactosemia and
their caregivers: disease burden and challenges with daily living. Orphanet J
Rare Dis. 2022 Mar 28;17(1):138. doi: 10.1186/s13023-022-02287-9. PMID:
35346295; PMCID: PMC8959560.