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Phase I safety and pharmacokinetic dose-escalation study of pilaralisib polymorph E, a phosphoinositide 3-kinase inhibitor in tablet formulation, in patients with solid tumors or lymphoma.
Bechter OE, Dumez H, Costermans J, Punie K, Hsu K, Dedieu JF, Ghuysen AF, Francesconi E, Sharma J, Liu L, Schöffski P. Bechter OE, et al. Among authors: sharma j. Cancer Chemother Pharmacol. 2016 Jul;78(1):83-90. doi: 10.1007/s00280-016-3056-0. Epub 2016 May 11. Cancer Chemother Pharmacol. 2016. PMID: 27169794 Clinical Trial.
A phase I dose-escalation study of the safety and pharmacokinetics of a tablet formulation of voxtalisib, a phosphoinositide 3-kinase inhibitor, in patients with solid tumors.
Mehnert JM, Edelman G, Stein M, Camisa H, Lager J, Dedieu JF, Ghuysen AF, Sharma J, Liu L, LoRusso PM. Mehnert JM, et al. Among authors: sharma j. Invest New Drugs. 2018 Feb;36(1):36-44. doi: 10.1007/s10637-017-0467-7. Epub 2017 Apr 17. Invest New Drugs. 2018. PMID: 28417284 Clinical Trial.
Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial.
Peterschmitt MJ, Saiki H, Hatano T, Gasser T, Isaacson SH, Gaemers SJM, Minini P, Saubadu S, Sharma J, Walbillic S, Alcalay RN, Cutter G, Hattori N, Höglinger GU, Marek K, Schapira AHV, Scherzer CR, Simuni T, Giladi N, Sardi SP, Fischer TZ; MOVES-PD Investigators. Peterschmitt MJ, et al. Among authors: sharma j. J Parkinsons Dis. 2022;12(2):557-570. doi: 10.3233/JPD-212714. J Parkinsons Dis. 2022. PMID: 34897099 Free PMC article. Clinical Trial.
Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.
Deegan PB, Goker-Alpan O, Geberhiwot T, Hopkin RJ, Lukina E, Tylki-Szymanska A, Zaher A, Sensinger C, Gaemers SJM, Modur V, Thurberg BL, Sharma J, Najafian B, Mauer M, DasMahapatra P, Wilcox WR, Germain DP. Deegan PB, et al. Among authors: sharma j. Mol Genet Metab. 2023 Feb;138(2):106963. doi: 10.1016/j.ymgme.2022.11.002. Epub 2022 Nov 9. Mol Genet Metab. 2023. PMID: 36481125 Free PMC article. Clinical Trial.
Atypical free sialic acid storage disorder associated with tissue specific mosaicism of SLC17A5.
Shinawi M, Wegner DJ, Paul AJ, Buchser W, Schmidt R, Sharma J, Sardiello M, Sisco K, Manwaring L, Reynolds M, Fulton R, Fronick C, Shaver A, Huang TY, Carroll A, Roessler K, Halpern AL; Undiagnosed Diseases Network; Dickson PI, Wambach JA. Shinawi M, et al. Among authors: sharma j. Mol Genet Metab. 2024 Dec 24;144(1):109004. doi: 10.1016/j.ymgme.2024.109004. Online ahead of print. Mol Genet Metab. 2024. PMID: 39742826
2,822 results