Publications

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Displaying 1 through 45 of 45 publications.

Mansfield C, Boeri M, Coulter J, Baranowski E, Sparks S, An Haack K, Hamed A. The value of knowing: preferences for genetic testing to diagnose rare muscle diseases. Orphanet J Rare Dis. 2024 Apr 22;19(1):173. doi: 10.1186/s13023-024-03160-7

Yee KS, Alexanderian D, Martin S, Olayinka-Amao B, Whiteman DAH. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment. Orphanet J Rare Dis. 2024 Apr 12;19(1):148. doi: 10.1186/s13023-024-03147-4

Yee KS, Lewis S, Evans E, Romano (DeMuro) C, Alexanderian D. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. Orphanet J Rare Dis. 2024 Mar 10;19(1):100. doi: 10.1186/s13023-024-03034-y

Koch S, Peralta GP, Carsin AE, Abellan A, Roda C, Torrent M, Iniguez C, Ballester F, Ferrero A, Zabaleta C, Lertxundi A, Guxens M, Vrijheid M, Sunyer J, Casas M, Garcia-Aymerich J. Physical activity and body mass related to catch-up lung function growth in childhood: a population-based accelerated cohort study. Thorax. 2024 Mar 6;79(8):762-9. doi: 10.1136/thorax-2022-219666

Carsin AE, Garcia-Aymerich J, Accordini S, Dharmage S, Leynaert B, de Las Heras M, Casas L, Caviezel S, Demoly P, Forsberg B, Gislason T, Corsico AG, Janson C, Jogi R, Martinez-Moratalla J, Nowak D, Gomez LP, Pin I, Probst-Hensch N, Raherison-Semjen C, Squillacioti G, Svanes C, Toren K, Urrutia I, Huerta I, Anto JM, Jarvis D, Guerra S. Spirometric patterns in young and middle-aged adults: a 20-year European study. Thorax. 2024 Jan 18;79(2):153-62. doi: 10.1136/thorax-2022-219696

Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2

Moitra S, Carsin AE, Abramson MJ, Accordini S, Amaral AFS, Anto J, Bono R, Casas Ruiz L, Cerveri I, Chatzi L, Demoly P, Dorado-Arenas S, Forsberg B, Gilliland F, Gislason T, Gullon JA, Heinrich J, Holm M, Janson C, Jogi R, Gomez Real F, Jarvis D, Leynaert B, Nowak D, Probst-Hensch N, Sanchez-Ramos JL, Raherison-Semjen C, Siroux V, Guerra S, Kogevinas M, Garcia-Aymerich J. Long-term effect of asthma on the development of obesity among adults: an international cohort study, ECRHS. Thorax. 2023 Feb;78(2):128-35. doi: 10.1136/thoraxjnl-2021-217867

Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4

Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z

Foreman PK, Margulis AV, Alexander K, Shediac R, Calingaert B, Harding A, Pladevall-Vila M, Landis S. Birth prevalence of phenylalanine hydroxylase deficiency: a systematic literature review and meta-analysis. Orphanet J Rare Dis. 2021 Jun 3;16(1):253. doi: 10.1186/s13023-021-01874-6

Lennon RP, Miller EL, Snyder B, Van Scoy LJ. Self-reported influenza and influenza-like symptoms in U.S. adults age 18-64 between September 1, 2019 and April 15, 2020. J Clin VIrol. 2021 Jan;134:104709. doi: 10.1016/j.jcv.2020.104709

Peralta GP, Marcon A, Carsin AE, Abramson MJ, Accordini S, Amaral AF, Anto JM, Bowatte G, Burney P, Corsico A, Demoly P, Dharmage S, Forsberg B, Fuertes E, Garcia-Larsen V, Gislason T, Gullon JA, Heinrich J, Holm M, Jarvis DL, Janson C, Jogi R, Johannessen A, Leynaert B, Rovira JM, Nowak D, Probst-Hensch N, Raherison C, Sanchez-Ramos JL, Sigsgaard T, Siroux V, Squillacioti G, Urrutia I, Weyler J, Zock JP, Garcia-Aymerich J. Body mass index and weight change are associated with adult lung function trajectories: the prospective ECRHS study. Thorax. 2020 Apr;75(4):313-20. doi: 10.1136/thoraxjnl-2019-213880

Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien Ansquer V, Germain DP, Lidove O. Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire. Orphanet J Rare Dis. 2019 Dec 4;14(1):284. doi: 10.1186/s13023-019-1254-7

Standaert B, Van Vlaenderen I, Van Bellinghen LA, Talbird SE, Hicks K, Carrico J, Buck PO. Constrained optimization for the selection of influenza vaccines to maximize the population benefit: a demonstration project. Appl Health Econ Health Policy. 2019 Nov 22. doi: 10.1007/s40258-019-00534-y

Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay H. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6

Harrington M, Whalley D, Twiss J, Rushton R, Martin S, Huynh L, Yang H. Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers. Orphanet J Rare Dis. 2019 Apr 29;14(1):89. doi: 10.1186/s13023-019-1060-2

Lytras T, Kogevinas M, Kromhout H, Carsin AE, Anto JM, Bentouhami H, Weyler J, Heinrich J, Nowak D, Urrutia I, Martinez-Moratalla J, Gullon JA, Pereira-Vega A, Raherison-Semjen C, Pin I, Demoly P, Leynaert B, Villani S, Gislason T, Svanes C, Holm M, Forsberg B, Norback D, Mehta AJ, Probst-Hensch N, Benke G, Jogi R, Toren K, Sigsgaard T, Schlunssen V, Olivieri M, Blanc PD, Vermeulen R, Garcia-Aymerich J, Jarvis D, Zock JP. Occupational exposures and 20-year incidence of COPD: the European Community Respiratory Health Survey. Thorax. 2018 Nov;73(11):1008-15. doi: 10.1136/thoraxjnl-2017-211158

Turner EE, Jenks M, McCool C, Marshall C, Millar L, Wood H, Peel A, Craig J, Sims AJ. The Memokath-051 stent for the treatment of ureteric obstruction: a NICE medical technology guidance. Appl Health Econ Health Policy. 2018 Aug;16(4):445-64. doi: 10.1007/s40258-018-0389-3

Fuertes E, Carsin AE, Anto JM, Bono R, Corsico AG, Demoly P, Gislason T, Gullon JA, Janson C, Jarvis D, Heinrich J, Holm M, Leynaert B, Marcon A, Martinez-Moratalla J, Nowak D, Pascual Erquicia S, Probst-Hensch NM, Raherison C, Raza W, Real FG, Russell M, Sanchez-Ramos JL, Weyler J, Garcia Aymerich J. Leisure-time vigorous physical activity is associated with better lung function: the prospective ECRHS study. Thorax. 2018 Apr;73(4):376-84. doi: 10.1136/thoraxjnl-2017-210947

Evans JM, Cleves A, Morgan H, Millar L, Carolan-Rees G. ENDURALIFE-powered cardiac resynchronisation therapy defibrillator devices for treating heart failure: a NICE Medical Technology Guidance. Appl Health Econ Health Policy. 2018 Apr;16(2):177-86. doi: 10.1007/s40258-017-0354-6.

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

Arbillaga-Etxarri A, Gimeno-Santos E, Barberan-Garcia A, Benet M, Borrell E, Dadvand P, Foraster M, Marin A, Monteagudo M, Rodriguez-Roisin R, Vall-Casas P, Vilaro J, Garcia-Aymerich J. Socio-environmental correlates of physical activity in patients with chronic obstructive pulmonary disease (COPD). Thorax. 2017 Sep;72(9):796-802. doi: 10.1136/thoraxjnl-2016-209209

Dancer RC, Parekh D, Lax S, D'Souza V, Zheng S, Bassford CR, Park D, Bartis DG, Mahida R, Turner AM, Sapey E, Wei W, Naidu B, Stewart PM, Fraser WD, Christopher KB, Cooper MS, Gao F, Sansom DM, Martineau AR, Perkins GD, Thickett DR. Vitamin D deficiency contributes directly to the Acute Respiratory Distress Syndrome (ARDS). Thorax. 2015 Jul;70(7):617-24. doi: 10.1136/thoraxjnl-2014-206680

Morales E, Garcia-Esteban R, de la Cruz OA, Basterrechea M, Lertxundi A, de Dicastillo MD, Zabaleta C, Sunyer J. Intrauterine and early postnatal exposure to outdoor air pollution and lung function at preschool age. Thorax. 2015 Jan;70(1):64-73. doi: 10.1136/thoraxjnl-2014-205413

Cai Y, Schikowski T, Adam M, Buschka A, Carsin AE, Jacquemin B, Marcon A, Sanchez M, Vierkotter A, Al-Kanaani Z, Beelen R, Birk M, Brunekreef B, Cirach M, Clavel-Chapelon F, Declercq C, de Hoogh K, de Nazelle A, Ducret-Stich RE, Valeria Ferretti V, Forsberg B, Gerbase MW, Hardy R, Heinrich J, Hoek G, Jarvis D, Keidel D, Kuh D, Nieuwenhuijsen MJ, Ragettli MS, Ranzi A, Rochat T, Schindler C, Sugiri D, Temam S, Tsai MY, Varraso R, Kauffmann F, Kramer U, Sunyer J, Kunzli N, Probst-Hensch N, Hansell AL. Cross-sectional associations between air pollution and chronic bronchitis: an ESCAPE meta-analysis across five cohorts. Thorax. 2014 Nov;69(11):1005-14. doi: 10.1136/thoraxjnl-2013-204352

Barbieri M, Weatherly HL, Ara R, Basarir H, Sculpher M, Adams R, Ahmed H, Coles C, Guerrero-Urbano T, Nutting C, Powell M. What is the quality of economic evaluations of non-drug therapies? A systematic review and critical appraisal of economic evaluations of radiotherapy for cancer. Appl Health Econ Health Policy. 2014 Oct;12(5):497-510. doi: 10.1007/s40258-014-0115-8

Karve S, Markowitz M, Fu DJ, Lindenmayer JP, Wang CC, Candrilli SD, Alphs L. Assessing medication adherence and healthcare utilization and cost patterns among hospital-discharged patients with schizoaffective disorder. Appl Health Econ Health Policy. 2014 Jun;12(3):335-46.

Meyers J, Yu Y, Kaye JA, Davis KL. Medicare fee-for-service enrollees with primary acute myeloid leukemia: an analysis of treatment patterns, survival, and healthcare resource utilization and costs. Appl Health Econ Health Policy. 2013 May 16;11(3):275-86. doi: 10.1007/s40258-013-0032-2

Karve S, Misurski D, Herrera-Taracena G, Davis KL. Annual all-cause healthcare costs among influenza patients with and without influenza-related complications: analysis of a United States managed care database. Appl Health Econ Health Policy. 2013 Apr 1;11(2):119-28.

Arvelo W, Sosa SM, Juliao P, Lopez MR, Estevez A, Lopez B, Morales-Betoulle ME, Gonzalez M, Gregoricus NA, Hall AJ, Vinje J, Parashar U, Lindblade KA. Norovirus outbreak of probable waterborne transmission with high attack rate in a Guatemalan resort. J Clin VIrol. 2012 Sep;55(1):8-11. doi: 10.1016/j.jcv.2012.02.018

Leynaert B, Sunyer J, Garcia-Esteban R, Svanes C, Jarvis D, Cerveri I, Dratva J, Gislason T, Heinrich J, Janson C, Kuenzli N, de Marco R, Omenaas E, Raherison C, Gomez Real F, Wjst M, Zemp E, Zureik M, Burney PG, Anto JM, Neukirch F. Gender differences in prevalence, diagnosis and incidence of allergic and non-allergic asthma: a population-based cohort. Thorax. 2012 Jul;67(7):625-31. doi: 10.1136/thoraxjnl-2011-201249

Kattan MW, Earnshaw SR, McDade CL, Black LK, Andriole GL. Cost effectiveness of chemoprevention for prostate cancer with dutasteride in a high-risk population based on results from the REDUCE clinical trial. Appl Health Econ Health Policy. 2011 Sep 1;9(5):305-15.

Varadhachary GR, Spector Y, Abbruzzese JL, Rosenwald S, Wang H, Aharonov R, Carlson HR, Cohen D, Karanth S, Macinskas J, Lenzi R, Chajut A, Edmonston TB, Raber MN. Prospective gene signature study using microRNA to identify the tissue of origin in patients with carcinoma of unknown primary. Clin Cancer Res. 2011 Jun 15;17(12):4063-70. doi: 10.1158/1078-0432.CCR-10-2599

Davis KL, Bell TJ, Miller JM, Misurski DA, Bapat B. Hospital costs, length of stay and mortality associated with childhood, adolescent and young adult meningococcal disease in the US. Appl Health Econ Health Policy. 2011 May 1;9(3):197-207.

Norback D, Zock JP, Plana E, Heinrich J, Svanes C, Sunyer J. Lung function decline in relation to mould and dampness in the home: the longitudinal European Community Respiratory Health Survey ECRHS II. Thorax. 2011 May;66(5):396-401.

Svanes C, Sunyer J, Plana E, Dharmage S, Heinrich J, Jarvis D. Early life origins of chronic obstructive pulmonary disease. Thorax. 2010 Jan;65(1):14-20.

Kunzli N, Bridevaux PO, Liu LJ, Garcia-Esteban R, Schindler C, Gerbase MW, Sunyer J, Keidel D, Rochat T, Swiss Cohort Study on Air Pollution and Lung Diseases in Adults. Traffic-related air pollution correlates with adult-onset asthma among never-smokers. Thorax. 2009 Aug;64(8):664-70. doi: 10.1136/thx.2008.110031

Earnshaw SR, Graham J, Oleen-Burkey M, Castelli-Haley J, Johnson K. Cost effectiveness of glatiramer acetate and natalizumab in relapsing-remitting multiple sclerosis. Appl Health Econ Health Policy. 2009 Jun 1;7(2):91-108.

Toren K, Zock JP, Kogevinas M, Plana E, Sunyer J, Radon K. An international prospective general population-based study of respiratory work disability. Thorax. 2009 Apr;64(4):339-44.

Davis KL, Mitra D, Kotapati S, Ibrahim R, Wolchok JD. Direct economic burden of high risk and metastatic melanoma: evidence from the SEER-Medicare linked database. Appl Health Econ Health Policy. 2009;7(1):31-41.

West R, Zhou X. Is nicotine replacement therapy for smoking cessation effective in the "real world"? Findings from a prospective multinational cohort study. Thorax. 2007 Nov 1;62(11):998-1002. doi: 10.1136/thx.2007.078758

Wenham RM, Schildkraut JM, McLean K, Calingaert B, Bentley RC, Marks J, Berchuck A. Polymorphisms in BRCA1 and BRCA2 and risk of epithelial ovarian cancer. Clin Cancer Res. 2003 Oct 1;9(12):4396-403.

LoRusso PM, Herbst RS, Rischin D, Ranson M, Calvert H, Raymond E, Kieback D, Kaye S, Gianni L, Harris A, Bjork T, Maddox A, Rothenberg ML, Small EJ, Rubin EH, Feyereislova A, Heyes A, Averbuch SD, Ochs J, Baselga J. Improvements in quality of life and disease-related symptoms in phase I trials of the selective oral epidermal growth factor receptor tyrosine kinase inhibitor ZD1839 in non-small cell lung cancer and other solid tumors. Clin Cancer Res. 2003 Jun 1;9(6):2040-8.

Lancaster JM, Sayer R, Blanchette C, Calingaert B, Whitaker R, Schildkraut J, Marks J, Berchuck A. High expression of tumor necrosis factor-related apoptosis-inducing ligand is associated with favorable ovarian cancer survival. Clin Cancer Res. 2003 Feb 1;9(2):762-6.

Doughty N, McKenna SP, Meads D, Doward LC, Pepke-Zaba J. Interim validation studies of new quality of life (QOL) and health-related quality of life (HRQL) measures specific to pulmonary arterial hypertension (PAH). Thorax. 2003;58(S3):84.