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101 results

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Page 1
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. Among authors: berger ki. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
Toscano A, Pollissard L, Msihid J, van der Beek N, Kishnani PS, Dimachkie MM, Berger KI, DasMahapatra P, Thibault N, Hamed A, Zhou T, Haack KA, Schoser B. Toscano A, et al. Among authors: berger ki. Mol Genet Metab. 2024 Feb;141(2):108121. doi: 10.1016/j.ymgme.2023.108121. Epub 2023 Dec 27. Mol Genet Metab. 2024. PMID: 38184428 Free article. Clinical Trial.
Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.
Boentert M, Berger KI, Díaz-Manera J, Dimachkie MM, Hamed A, Riou França L, Thibault N, Shukla P, Ishak J, Caro JJ. Boentert M, et al. Among authors: berger ki. Orphanet J Rare Dis. 2024 Jan 12;19(1):14. doi: 10.1186/s13023-023-02974-1. Orphanet J Rare Dis. 2024. PMID: 38216959 Free PMC article.
Pompe disease diagnosis and management guideline.
Kishnani PS, Steiner RD, Bali D, Berger K, Byrne BJ, Case LE, Crowley JF, Downs S, Howell RR, Kravitz RM, Mackey J, Marsden D, Martins AM, Millington DS, Nicolino M, O'Grady G, Patterson MC, Rapoport DM, Slonim A, Spencer CT, Tifft CJ, Watson MS. Kishnani PS, et al. Genet Med. 2006 May;8(5):267-88. doi: 10.1097/01.gim.0000218152.87434.f3. Genet Med. 2006. PMID: 16702877 Free PMC article. No abstract available.
Lower Airway Dysbiosis Augments Lung Inflammatory Injury in Mild-to-Moderate Chronic Obstructive Pulmonary Disease.
Sulaiman I, Wu BG, Chung M, Isaacs B, Tsay JJ, Holub M, Barnett CR, Kwok B, Kugler MC, Natalini JG, Singh S, Li Y, Schluger R, Carpenito J, Collazo D, Perez L, Kyeremateng Y, Chang M, Campbell CD, Hansbro PM, Oppenheimer BW, Berger KI, Goldring RM, Koralov SB, Weiden MD, Xiao R, D'Armiento J, Clemente JC, Ghedin E, Segal LN. Sulaiman I, et al. Among authors: berger ki. Am J Respir Crit Care Med. 2023 Nov 15;208(10):1101-1114. doi: 10.1164/rccm.202210-1865OC. Am J Respir Crit Care Med. 2023. PMID: 37677136
Role of small airway dysfunction in unexplained exertional dyspnoea.
Sharpe AL, Reibman J, Oppenheimer BW, Goldring RM, Liu M, Shao Y, Bohart I, Kwok B, Weinstein T, Addrizzo-Harris D, Sterman DH, Berger KI. Sharpe AL, et al. Among authors: berger ki. ERJ Open Res. 2023 Jun 5;9(3):00603-2022. doi: 10.1183/23120541.00603-2022. eCollection 2023 May. ERJ Open Res. 2023. PMID: 37284422 Free PMC article.
Computer clinical decision support that automates personalized clinical care: a challenging but needed healthcare delivery strategy.
Morris AH, Horvat C, Stagg B, Grainger DW, Lanspa M, Orme J, Clemmer TP, Weaver LK, Thomas FO, Grissom CK, Hirshberg E, East TD, Wallace CJ, Young MP, Sittig DF, Suchyta M, Pearl JE, Pesenti A, Bombino M, Beck E, Sward KA, Weir C, Phansalkar S, Bernard GR, Thompson BT, Brower R, Truwit J, Steingrub J, Hiten RD, Willson DF, Zimmerman JJ, Nadkarni V, Randolph AG, Curley MAQ, Newth CJL, Lacroix J, Agus MSD, Lee KH, deBoisblanc BP, Moore FA, Evans RS, Sorenson DK, Wong A, Boland MV, Dere WH, Crandall A, Facelli J, Huff SM, Haug PJ, Pielmeier U, Rees SE, Karbing DS, Andreassen S, Fan E, Goldring RM, Berger KI, Oppenheimer BW, Ely EW, Pickering BW, Schoenfeld DA, Tocino I, Gonnering RS, Pronovost PJ, Savitz LA, Dreyfuss D, Slutsky AS, Crapo JD, Pinsky MR, James B, Berwick DM. Morris AH, et al. Among authors: berger ki. J Am Med Inform Assoc. 2022 Dec 13;30(1):178-194. doi: 10.1093/jamia/ocac143. J Am Med Inform Assoc. 2022. PMID: 36125018 Free PMC article.
Molecular Clustering Analysis of Blood Biomarkers in World Trade Center Exposed Community Members with Persistent Lower Respiratory Symptoms.
Grunig G, Durmus N, Zhang Y, Lu Y, Pehlivan S, Wang Y, Doo K, Cotrina-Vidal ML, Goldring R, Berger KI, Liu M, Shao Y, Reibman J. Grunig G, et al. Among authors: berger ki. Int J Environ Res Public Health. 2022 Jul 1;19(13):8102. doi: 10.3390/ijerph19138102. Int J Environ Res Public Health. 2022. PMID: 35805759 Free PMC article.
101 results