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The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): a phase II, multicentre, open-label, screening study.
Burton BK, Grange DK, Milanowski A, Vockley G, Feillet F, Crombez EA, Abadie V, Harding CO, Cederbaum S, Dobbelaere D, Smith A, Dorenbaum A. Burton BK, et al. Among authors: dorenbaum a. J Inherit Metab Dis. 2007 Oct;30(5):700-7. doi: 10.1007/s10545-007-0605-z. Epub 2007 Sep 12. J Inherit Metab Dis. 2007. PMID: 17846916 Clinical Trial.
Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial.
Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S. Longo N, et al. Among authors: dorenbaum a. Lancet. 2014 Jul 5;384(9937):37-44. doi: 10.1016/S0140-6736(13)61841-3. Epub 2014 Apr 14. Lancet. 2014. PMID: 24743000 Free PMC article. Clinical Trial.
Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study.
Gonzales E, Hardikar W, Stormon M, Baker A, Hierro L, Gliwicz D, Lacaille F, Lachaux A, Sturm E, Setchell KDR, Kennedy C, Dorenbaum A, Steinmetz J, Desai NK, Wardle AJ, Garner W, Vig P, Jaecklin T, Sokal EM, Jacquemin E. Gonzales E, et al. Among authors: dorenbaum a. Lancet. 2021 Oct 30;398(10311):1581-1592. doi: 10.1016/S0140-6736(21)01256-3. Epub 2021 Oct 28. Lancet. 2021. PMID: 34755627 Clinical Trial.
Maralixibat for the treatment of PFIC: Long-term, IBAT inhibition in an open-label, Phase 2 study.
Loomes KM, Squires RH, Kelly D, Rajwal S, Soufi N, Lachaux A, Jankowska I, Mack C, Setchell KDR, Karthikeyan P, Kennedy C, Dorenbaum A, Desai NK, Garner W, Jaecklin T, Vig P, Miethke A, Thompson RJ. Loomes KM, et al. Among authors: dorenbaum a. Hepatol Commun. 2022 Sep;6(9):2379-2390. doi: 10.1002/hep4.1980. Epub 2022 May 4. Hepatol Commun. 2022. PMID: 35507739 Free PMC article. Clinical Trial.
A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis.
Mayo MJ, Pockros PJ, Jones D, Bowlus CL, Levy C, Patanwala I, Bacon B, Luketic V, Vuppalanchi R, Medendorp S, Dorenbaum A, Kennedy C, Novak P, Gu J, Apostol G, Hirschfield GM. Mayo MJ, et al. Among authors: dorenbaum a. Hepatol Commun. 2019 Feb 1;3(3):365-381. doi: 10.1002/hep4.1305. eCollection 2019 Mar. Hepatol Commun. 2019. PMID: 30859149 Free PMC article.
Safety, tolerability and pharmacodynamics of apical sodium-dependent bile acid transporter inhibition with volixibat in healthy adults and patients with type 2 diabetes mellitus: a randomised placebo-controlled trial.
Tiessen RG, Kennedy CA, Keller BT, Levin N, Acevedo L, Gedulin B, van Vliet AA, Dorenbaum A, Palmer M. Tiessen RG, et al. Among authors: dorenbaum a. BMC Gastroenterol. 2018 Jan 5;18(1):3. doi: 10.1186/s12876-017-0736-0. BMC Gastroenterol. 2018. PMID: 29304731 Free PMC article. Clinical Trial.
46 results