Publications

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Displaying 51 through 88 of 88 publications.

Ayodele O, Parikh RC, Ajmera A, Esterberg E, Desai NK, Williams J, Katzka DA. Assessing the diagnostic journey, burden of disease, and treatment patterns in patients with eosinophilic esophagitis in a real world setting. Poster presented at the ACG 2020 Virtual Annual Scientific Meeting; October 23, 2020.

Ayodele O, Parikh RC, Esterberg E, Ajmera M, Goodwin B, Desai NK, Williams J, Katzka DA. Characterizing the burden of disease in younger children, adults, and older adults with eosinophilic esophagitis in a real world setting. Poster presented at the ACG 2020 Virtual Annual Scientific Meeting; October 23, 2020.

Mader G, Purser MF, Mladsi DM, Sanon M, Oberdhan D, Watnick TJ, Seliger SL. Benefit of tolvaptan on time to ESRD for patients with rapidly progressing autosomal dominant polycystic kidney disease (ADPKD): a disease progression model. Poster presented at the 2020 Virtual American Society of Nephrology Kidney Week Conference; October 22, 2020. [abstract] J Am Soc Nephrol. 2020 Oct 22; 31:492.

Mader G, Mladsi DM, Zhou X, Sanon M, Wang J, Willey CJ, Seliger SL. Mortality risk in elderly ESRD patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD): study findings using data from the United States Renal Data System. Poster presented at the 2020 Virtual American Society of Nephrology Kidney Week Conference; October 22, 2020. [abstract] J Am Soc Nephrol. 2020 Oct; 31:503-4.

Ueda K, Takura T, Fujikoshi S, Meyers J, Nagar SP, Enomoto H. Longitudinal assessment of pain management among the employed Japanese population with knee osteoarthritis. Clin Interv Aging. 2020 Jun 26;2020(15):1003-12. doi: 10.2147/CIA.S242083

Copley-Merriman C, Yang X, Juniper M, Amin S, Yoo HK, Sen S. Impact of neurofibromatosis type 1 and plexiform neurofibromas on patient-reported health-related quality of life. Poster presented at the 2020 ISPOR Virtual Conference; May 2020. [abstract] Value Health. 2020 May 1; 23(Suppl 1):S344. doi: 10.1016/j.jval.2020.04.371

Mansfield C, Boeri M, Coulter J, Baranowski E, Sparks S, Hamed A. Preferences for genetic tests: trading off speed of diagnosis, availability of treatment, and cost. Poster presented at the 2020 ISPOR Virtual Conference; May 2020. [abstract] Value Health. 2020 May 1; 23(Suppl 1):S345. doi: 10.1016/j.jval.2020.04.094

Purser M, Gallagher M, Mladsi D, Weber JM, Andermariam B, Kaye JA, Chawla A. Development of key criteria for an economic model for a potentially curative one-time treatment for sickle cell disease and evaluation of published models with respect to these criteria. Hemasphere. 2020;p.1098-9.

Kabadi SM, Goyal RK, Nagar SP, Davis KL, Le H, Du XL, Jain P, Wang ML, Romaguera JE, Kaye JA. Overall survival, adverse events, and economic burden in Medicare-insured patients with mantle cell lymphoma receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 7, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13; 134(Supplement_1):63. doi: 10.1182/blood-2019-122841

Goyal RK, Nagar SP, Kabadi SM, Davis KL, Le H, Kaye JA. Overall survival, adverse events, and economic burden in Medicare patients with chronic lymphocytic leukemia receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 9, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13; 134(Supplement 1):796. doi: 10.1182/blood-2019-122842

Ueda K, Takura T, Fujikoshi S, Meyers J, Nagar SP, Enomoto H. A claims database longitudinal study of the economic burden among patients with knee osteoarthritis in Japan. Poster presented at the 34th Annual Research Meeting of the Japanese Orthopaedic Association; October 17, 2019. Yokohama, Japan.

Martin S, Chen K, Harris N, Vera-Llonch M, Krasner A. Development of a patient-reported outcome measure for chronic hypoparathyroidism. Adv Ther. 2019 Aug;36(8):1999-2009. doi: 10.1007/s12325-019-00999-2

Harrington M, Hareendran A, Skalicky A, Wilson H, Clark M, Mikl J. Assessing the impact on caregivers caring for patients with rare pediatric lysosomal storage diseases: development of the Caregiver Impact Questionnaire. J Patient Rep Outcomes. 2019 Jul 23;3(1):44. doi: 10.1186/s41687-019-0140-3

Mader G, Purser M, Mladsi D, Sanon M, Oberdhan D, Watnick T, Seliger S. Modeling disease progression in patients at risk of rapid progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD): using the mayo classification prediction equation. Poster presented at the 2019 National Kidney Foundation Spring Clinical Meeting; May 9, 2019. Boston, MA.

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

Clark M, Berry P, Martin S, Harris N, Sprecher D, Olitsky S, Hoag JB. Nosebleeds in hereditary hemorrhagic telangiectasia: development of a patient-completed daily eDiary. Laryngoscope Investig Otolaryngol. 2018 Nov 14;3(6):439-45. doi: 10.1002/lio2.211

Long J, Hunter S, Banerji A, Jain G, Hollis K, Brown TM, Devercelli G, Davis K. Burden of illness in hereditary angioedema: findings from a US patient survey. Presented at the HAE Global Conference 2018; May 18, 2018. Vienna, Austria.

Raspa M, Sacco P, Bailey DB, Visootsak J, Cabo R. Clinical unmet needs and burden in Fragile X syndrome: results of a targeted literature review. Poster presented at the 2018 AMCP Managed Care & Specialty Pharmacy Annual Meeting; April 23, 2018. Boston, MA. [abstract] J Manag Care Pharm. 2018 Apr 24; 24(4-a):S7. Previously presented at the American Association on Intellectual and Development Disabilities 141st Annual Meeting.

P Sacco, Raspa M, Leahy L, Cabo R. A literature review of the economic burden of Fragile X Syndrome. Poster presented at the 70th AAN Annual Meeting; April 24, 2018. Los Angeles, CA. [abstract] Neurology. 2018 Apr 10; 90(15 Supplement):3.319.

Krishnarajah G, Knox H, Graham CN, Christiansen S, Zuraw B. Subcutaneous C1-inhibitor prophylactic treatment for hereditary angioedema: a budget impact model. Poster presented at the 2018 AMCP Managed Care & Specialty Pharmacy Annual Meeting; April 23, 2018. Boston, MA. [abstract] J Manag Care Pharm. 2018 Apr; 24(4-a):S37.

Sacco P, Raspa M, Leahy L, Cabo R. A literature review of the economic burden of Fragile X syndrome. Poster presented at the 2018 AMCP Managed Care & Specialty Pharmacy Annual Meeting; April 23, 2018. Boston, MA. [abstract] J Manag Care Pharm. 2018 Apr; 24(4-a):S7. Previously presented at the 70th AAN Annual Meeting.

Devercelli G, Davis K, Brown M, Hollis K, Hunter S, Long J, Jain G, Banerji A. Burden of hereditary angioedema: findings from a US patient survey. Poster presented at the 2018 AAAAI/WAO Joint Congress; March 3, 2018. Orlando, FL. [abstract] J Allergy Clin Immunol. 2018 Feb; 141(2 Suppl):AB57. doi: 10.1016/j.jaci.2017.12.182

Wheeler AC, Sacco P, Cabo R. Unmet clinical needs and burden in Angelman syndrome: a review of the literature. Orphanet J Rare Dis. 2017 Oct 16;12(1):164. doi: 10.1186/s13023-017-0716-z

Banerji A, Davis KH, Devercelli G, Hollis K, Hunter S, Jain G. Clinical and demographic characteristics of patients with hereditary angioedema in the United States. Poster presented at the American College of Allergy, Asthma, and Immunology 2017 Annual Scientific Meeting; October 2017. Boston, MA.

Raspa M, Sacco P, Bailey DB, Visootak J, Cabo R. Clinical unmet needs and burden in fragile X syndrome: results of a targeted literature review. Poster presented at the American Association on Intellectual and Development Disabilities 141st Annual Meeting; June 27, 2017. Hartford, CT.

Wheeler A, Sacco P, Rakhit A, Cabo R. Identifying the unmet medical need of Angelman Syndrome: results of a targeted literature review. Poster presented at the 2016 ISPOR 19th Annual European Congress; November 1, 2016. Vienna, Austria. [abstract] Value Health. 2016 Nov; 19(7):A579.

Williams V, Coles T, Gnanasakthy A, DeMuro C, Yarr S, Williams NJ, Lowes L, Alfano L, Tseng B. Psychometric validation of a patient-reported measure of physical functioning in sporadic inclusion body myositis. Muscle Nerve. 2016 Oct 13;54(4):658-65. doi: 10.1002/mus.25080

DeMuro C, Lewis S, Lowes L, Alfano L, Tseng B, Gnanasakthy A. Development of the sporadic inclusion body myositis physical functioning assessment (sIFA). Muscle Nerve. 2016 Oct;54(4):653-7. doi: 10.1002/mus.25079

Bell C, Kurosky SK, Candrilli SD. Muscular dystrophy-related hospitalizations among male pediatric patients in the United States. Hospital Practice. 2015 Aug;43(3):180-5.

Williams V, Coles T, DeMuro C, Lewis S, Williams N, Yarr S, Barghout V, Lowes L, Alfano L, Goldberg B, Gnanasakthy A, Tseng B. Psychometric evaluation of the Sporadic Inclusion Body Myositis (sIBM) Physical Functioning Assessment (sIFA). Poster presented at the 1st Annual International Conference on Myositis; May 2015. Stockholm, Sweden.

DeMuro C, Lewis S, Lowes L, Tseng B, Gnanasakthy A. A novel measure to assess self-reported physical functioning in patients with sporadic inclusion body myositis. Poster presented at the 2015 ISPOR 20th Annual International Meeting; May 2015. Philadelphia, PA.

Barghout V, DeMuro C, Price M, Lewis S, Tseng B. Skeletal muscle activity and resource tool for sporadic inclusion body myositis: characterization of resource utilization and financial burden experienced by sIBM patients. Poster presented at the 2015 ISPOR 20th Annual International Meeting; May 2015. Philadelphia, PA.

Gnanasakthy A, DeMuro C, Lewis S, Tseng B. Development of a disease model for sporadic inclusion body myositis. Poster presented at the 2015 ISPOR 20th Annual International Meeting; May 2015. Philadelphia, PA.

Fehnel S, Brown TM, Nelson L, Chen A, Roof E, Kim DD, Dykens EM. Development of the hyperphagia questionnaire for use in prader-willi syndrome clinical trials. Poster presented at the 2015 ISPOR 20th Annual International Meeting; May 2015. Philadelphia, PA.

Webb S, Ware JE, Forsythe A, Yang M, Badia X, Nelson LM, Signorovitch JE, McLeod L, Maldonado M, Zgliczylski W, De Block C, Portocarrero-Ortiz L, Gadelha MR. Treatment effectiveness of pasireotide on health-related quality of life in patients with Cushing's disease. Eur J Endocrinol. 2014 Jul;171(1):89-98. doi: 10.1530/EJE-13-1013

Nelson LM, Forsythe A, McLeod L, Pulgar S, Maldonado M, Coles T, Zhang Y, Webb SM, Badia X. Psychometric evaluation of the Cushing's Quality-of-Life Questionnaire. Patient. 2013 Apr 1;Advance Online.

Badia X, Forsythe A, Nelson LM, Coles TM, McLeod LD, Webb SM. Improvements in health-related quality of life in acromegaly with pasireotide LAR and octreotide LAR: results from a large, randomized, double-blind phase III trial. Poster presented at the 2012 ISPOR 15th Annual European Congress; November 2012. Berlin, Germany. [abstract] Value Health. 2012 Nov; 15(7):A505.

Badia X, Roset M, Forsythe A, Coles TM, McLeod LD, Nelson LM. Clinical and patient-reported outcomes associated to pasireotide in patients with Cushing's disease. Poster presented at the 15th Annual Congress of the European Neuroendocrine Association; September 2012.