Publications

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Displaying 1 through 9 of 9 publications.

O'Neill C, Mansfield C, Drake E, Coulter J, Peay HL. Parent prioritization of meaningful treatment targets for Sanfilippo syndrome. Poster presented at the 2020 World Symposium; February 2020. Orlando, FL. [abstract] Mol Genet Metab. 2020 Feb; 129(2):S121. doi: 10.1016/j.ymgme.2019.11.315

Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien V, Germain DP, Lidove O. Fabry patients' needs and expectations regarding their treatment in France: development of a Patients' Need Questionnaire (PNQ Fabry). Poster presented at the 2019 WORLDSymposium; February 2019. Orlando, FL. [abstract] Mol Genet Metab. 2019 Feb; 126(2):S110. doi: 10.1016/j.ymgme.2018.12.278

Kreher N, Phillips D, Fehnel S, Brown M. Evaluating the content validity of the Diary of Irritable Bowel Syndrome Symptoms – Mixed (DIBSS-M) to assess gastrointestinal symptoms associated with Fabry disease. Poster presented at the 16th Annual WORLDSymposium; February 11, 2019. Orlando, FL. [abstract] Mol Genet Metab. 2019 Feb; 126(2):S87. doi: 10.1016/j.ymgme.2018.12.215

Slota C, Quintana M, Shrader J, Joe G, McKew J, Fitzgerald M, Gahl WA, Berry S, Carrillo N. GNE myopathy: disease progression determined by longitudinal natural history data and mathematical modeling. Poster presented at the Society for Inherited Metabolic Disorders Conference; April 2016. Ponte Vedra Beach, FL. [abstract] Mol Genet Metab. 2016 Apr; 117(2016):221-98.

Pavel M, Horsch D, Anthony L, Ervin C, Kulke MH, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Lapuerta P. Patient interviews in TELESTAR, a phase 3 study of telotristat etiprate, report meaningful improvement in carcinoid syndrome. Poster presented at the 2016 European Neuroendocrine Tumor Society (ENETS) Annual Meeting; March 2016. Barcelona, Spain. [abstract] Neuroendocrinology. 2016; 103:89.

Lanza FL, Sontag SJ, Ciociola AA, Sykes DL, Heath AT, McSorley DJ. Ranitidine bismuth citrate plus clarithromycin: a dual therapy regimen for patients with duodenal ulcer. Helicobacter. 1998 Sep;3(3):212-21.