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Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.
Jones SA, Rojas-Caro S, Quinn AG, Friedman M, Marulkar S, Ezgu F, Zaki O, Gargus JJ, Hughes J, Plantaz D, Vara R, Eckert S, Arnoux JB, Brassier A, Le Quan Sang KH, Valayannopoulos V. Jones SA, et al. Orphanet J Rare Dis. 2017 Feb 8;12(1):25. doi: 10.1186/s13023-017-0587-3. Orphanet J Rare Dis. 2017. PMID: 28179030 Free PMC article. Clinical Trial.
Prevalence of heart failure with preserved ejection fraction in Latin American, Middle Eastern, and North African Regions in the I PREFER study (Identification of Patients With Heart Failure and PREserved Systolic Function: an epidemiological regional study).
Magaña-Serrano JA, Almahmeed W, Gomez E, Al-Shamiri M, Adgar D, Sosner P, Herpin D; I PREFER Investigators. Magaña-Serrano JA, et al. Am J Cardiol. 2011 Nov 1;108(9):1289-96. doi: 10.1016/j.amjcard.2011.06.044. Am J Cardiol. 2011. PMID: 22000627