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Jones-Waldman syndrome: another report?
Schuler L, Puga AC, Schwartz IV, Netto C. Schuler L, et al. Among authors: puga ac. Am J Med Genet. 1994 May 15;51(1):83. doi: 10.1002/ajmg.1320510118. Am J Med Genet. 1994. PMID: 8030675 No abstract available.
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome).
Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. Muenzer J, et al. Among authors: puga ac. Genet Med. 2006 Aug;8(8):465-73. doi: 10.1097/01.gim.0000232477.37660.fb. Genet Med. 2006. PMID: 16912578 Free article. Clinical Trial.
Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology.
Kaddi CD, Niesner B, Baek R, Jasper P, Pappas J, Tolsma J, Li J, van Rijn Z, Tao M, Ortemann-Renon C, Easton R, Tan S, Puga AC, Schuchman EH, Barrett JS, Azer K. Kaddi CD, et al. Among authors: puga ac. CPT Pharmacometrics Syst Pharmacol. 2018 Jul;7(7):442-452. doi: 10.1002/psp4.12304. Epub 2018 Jun 19. CPT Pharmacometrics Syst Pharmacol. 2018. PMID: 29920993 Free PMC article.
Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.
Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Ross L, Angell J, Puga AC. Cox TM, et al. Among authors: puga ac. Lancet. 2015 Jun 13;385(9985):2355-62. doi: 10.1016/S0140-6736(14)61841-9. Epub 2015 Mar 26. Lancet. 2015. PMID: 25819691 Clinical Trial.
25 results