Beck M - Search Results

1

The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I.

Pastores GM, Arn P, Beck M, Clarke JT, Guffon N, Kaplan P, Muenzer J, Norato DY, Shapiro E, Thomas J, Viskochil D, Wraith JE. Pastores GM, et al. Among authors: beck m. Mol Genet Metab. 2007 May;91(1):37-47. doi: 10.1016/j.ymgme.2007.01.011. Epub 2007 Mar 2. Mol Genet Metab. 2007. PMID: 17336562

2

Childhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I.

Thomas JA, Beck M, Clarke JT, Cox GF. Thomas JA, et al. Among authors: beck m. J Inherit Metab Dis. 2010 Aug;33(4):421-7. doi: 10.1007/s10545-010-9113-7. Epub 2010 Jun 2. J Inherit Metab Dis. 2010. PMID: 20532982 Free PMC article.

3

The natural history of MPS I: global perspectives from the MPS I Registry.

Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, Fallet S. Beck M, et al. Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27. Genet Med. 2014. PMID: 24675674 Free PMC article.

4

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.

Harmatz P, Giugliani R, D Schwartz IV, Guffon N, Teles EL, Miranda MCS, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Yu ZF, Swiedler SJ, Decker C; MPS VI Study Group. Harmatz P, et al. Among authors: beck m. Mol Genet Metab. 2008 Aug;94(4):469-475. doi: 10.1016/j.ymgme.2008.04.001. Epub 2008 May 23. Mol Genet Metab. 2008. PMID: 18502162 Clinical Trial.

5

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. Among authors: beck m. Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847. Pediatrics. 2009. PMID: 19117887 Clinical Trial.

6

Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase).

Wraith JE, Beck M, Lane R, van der Ploeg A, Shapiro E, Xue Y, Kakkis ED, Guffon N. Wraith JE, et al. Among authors: beck m. Pediatrics. 2007 Jul;120(1):e37-46. doi: 10.1542/peds.2006-2156. Epub 2007 Jun 4. Pediatrics. 2007. PMID: 17606547 Clinical Trial.

7

Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome).

Martin R, Beck M, Eng C, Giugliani R, Harmatz P, Muñoz V, Muenzer J. Martin R, et al. Among authors: beck m. Pediatrics. 2008 Feb;121(2):e377-86. doi: 10.1542/peds.2007-1350. Pediatrics. 2008. PMID: 18245410 Review.

8

Initial report from the Hunter Outcome Survey.

Wraith JE, Beck M, Giugliani R, Clarke J, Martin R, Muenzer J; HOS Investigators. Wraith JE, et al. Among authors: beck m. Genet Med. 2008 Jul;10(7):508-16. doi: 10.1097/gim.0b013e31817701e6. Genet Med. 2008. PMID: 18580692

9

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. Among authors: beck m. 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

10

Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey.

Muenzer J, Beck M, Giugliani R, Suzuki Y, Tylki-Szymanska A, Valayannopoulos V, Vellodi A, Wraith JE. Muenzer J, et al. Among authors: beck m. Genet Med. 2011 Feb;13(2):102-9. doi: 10.1097/GIM.0b013e318206786f. Genet Med. 2011. PMID: 21233716 Free article.

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