Clarke LA - Search Results

1

Enzyme-replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up.

Gabrielli O, Clarke LA, Bruni S, Coppa GV. Gabrielli O, et al. Among authors: clarke la. Pediatrics. 2010 Jan;125(1):e183-7. doi: 10.1542/peds.2009-1728. Epub 2009 Dec 21. Pediatrics. 2010. PMID: 20026495

2

12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment.

Gabrielli O, Clarke LA, Ficcadenti A, Santoro L, Zampini L, Volpi N, Coppa GV. Gabrielli O, et al. Among authors: clarke la. BMC Med Genet. 2016 Mar 10;17:19. doi: 10.1186/s12881-016-0284-4. BMC Med Genet. 2016. PMID: 26965916 Free PMC article.

3

Development of a mnemonic screening tool for identifying subjects with Hunter syndrome.

Cohn GM, Morin I, Whiteman DA; Hunter Outcome Survey Investigators. Cohn GM, et al. Eur J Pediatr. 2013 Jul;172(7):965-70. doi: 10.1007/s00431-013-1967-x. Epub 2013 Mar 7. Eur J Pediatr. 2013. PMID: 23468122 Free PMC article.

4

Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS).

Jones SA, Almássy Z, Beck M, Burt K, Clarke JT, Giugliani R, Hendriksz C, Kroepfl T, Lavery L, Lin SP, Malm G, Ramaswami U, Tincheva R, Wraith JE; HOS Investigators. Jones SA, et al. J Inherit Metab Dis. 2009 Aug;32(4):534-43. doi: 10.1007/s10545-009-1119-7. Epub 2009 Jul 14. J Inherit Metab Dis. 2009. PMID: 19597960

5

Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey.

Mendelsohn NJ, Harmatz P, Bodamer O, Burton BK, Giugliani R, Jones SA, Lampe C, Malm G, Steiner RD, Parini R; Hunter Outcome Survey Investigators. Mendelsohn NJ, et al. Genet Med. 2010 Dec;12(12):816-22. doi: 10.1097/GIM.0b013e3181f6e74d. Genet Med. 2010. PMID: 21045710 Free article.

6

Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Sidman M, Kakkis ED, Cox GF. Clarke LA, et al. Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847. Pediatrics. 2009. PMID: 19117887 Clinical Trial.

7

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Swiedler SJ, Kakkis ED, Braakman T, Chadbourne E, Walton-Bowen K, Cox GF. Wraith JE, et al. Among authors: clarke la. J Pediatr. 2004 May;144(5):581-8. doi: 10.1016/j.jpeds.2004.01.046. J Pediatr. 2004. PMID: 15126990 Clinical Trial.

8

Mucopolysaccharidosis I: management and treatment guidelines.

Muenzer J, Wraith JE, Clarke LA; International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Muenzer J, et al. Among authors: clarke la. Pediatrics. 2009 Jan;123(1):19-29. doi: 10.1542/peds.2008-0416. Pediatrics. 2009. PMID: 19117856

9

The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus.

Muenzer J, Bodamer O, Burton B, Clarke L, Frenking GS, Giugliani R, Jones S, Rojas MV, Scarpa M, Beck M, Harmatz P. Muenzer J, et al. Eur J Pediatr. 2012 Jan;171(1):181-8. doi: 10.1007/s00431-011-1606-3. Epub 2011 Oct 29. Eur J Pediatr. 2012. PMID: 22037758 Free PMC article.

10

Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure.

de Ru MH, Teunissen QG, van der Lee JH, Beck M, Bodamer OA, Clarke LA, Hollak CE, Lin SP, Rojas MV, Pastores GM, Raiman JA, Scarpa M, Treacy EP, Tylki-Szymanska A, Wraith JE, Zeman J, Wijburg FA. de Ru MH, et al. Among authors: clarke la. Orphanet J Rare Dis. 2012 Apr 23;7:22. doi: 10.1186/1750-1172-7-22. Orphanet J Rare Dis. 2012. PMID: 22524701 Free PMC article.

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