Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI

Molecular Genetics and Metabolism

Volumn 109, Issue 1, 2013, Pages 62-69

  Horovitz, Dafne D G ^a,q   Magalhães, Tatiana S P C ^a   Acosta, Angelina ^b   Ribeiro, Erlane M ^c   Giuliani, Liane R ^d   Palhares, Durval B ^d   Kim, Chong A ^e   De Paula, Ana Carolina ^e   Kerstenestzy, Marcelo ^f   Pianovski, Mara A D ^g   Costa, Maria Ione F ^h   Santos, Francisca C ⁱ   Martins, Ana Maria ^j   Aranda, Carolina S ^j   Neto, Jordão Correa ^k   Holanda, Gervina Brady Moreira ^l   Cardoso, Laércio ^b   da Silva, Carlos A B ^l,m   Bonatti, Renata C F ⁿ   Ribeiro, Bethania F R ^o   Rodrigues, Maria do Carmo S ^p   Llerena, Juan C ^a   more..

^a INSTITUTO OSWALDO CRUZ   (Brazil)

^b FEDERAL UNIVERSITY OF BAHIA   (Brazil)

^c Albert Sabin Hospital   (Brazil)

^d FEDERAL UNIVERSITY OF MATO GROSSO DO SUL   (Brazil)

^e UNIVERSITY OF SÃO PAULO   (Brazil)

^f HOSPITAL BARÃO DE LUCENA   (Brazil)

^g FEDERAL UNIVERSITY OF PARANÁ   (Brazil)

^h Centro de Reabilitação Infantil   (Brazil)

ⁱ Hospital Universitário do Maranhão   (Brazil)

^j FEDERAL UNIVERSITY OF SÃO PAULO   (Brazil)

^k PONTIFÍCIA UNIVERSIDADE CATÓLICA DE CAMPINAS   (Brazil)

^l FEDERAL UNIVERSITY OF RIO GRANDE DO NORTE   (Brazil)

^m UNIVERSITY OF FORTALEZA   (Brazil)

ⁿ FEDERAL UNIVERSITY OF TRIÂNGULO MINEIRO   (Brazil)

^o Hospital das Clínicas do Acre   (Brazil)

^p FEDERAL UNIVERSITY OF ESPÍRITO SANTO   (Brazil)

^q not available   (Brazil)

more..

Author keywords

Enzyme replacement therapy; Galsulfase; Lysosomal storage disorder; MPS VI; Mucopolysaccharidosis VI; Pediatric

Indexed keywords

GALSULFASE;

ARTICLE; CARDIOVASCULAR DISEASE; CENTRAL NERVOUS SYSTEM DISEASE; CHILD; CHILL; CLINICAL ARTICLE; COUGHING; CYANOSIS; DISEASE COURSE; DRUG EFFICACY; DRUG URINE LEVEL; ENZYME REPLACEMENT; EYE DISEASE; FEVER; GROUPS BY AGE; HEARING DISORDER; HUMAN; HYPERTENSION; MAROTEAUX LAMY SYNDROME; NAUSEA; NERVE CONDUCTION; PRESCHOOL CHILD; PRIORITY JOURNAL; RASH; SLEEP DISORDERED BREATHING; SPEECH DISORDER; TACHYCARDIA; TREATMENT OUTCOME;

EID: 84876092083     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.02.014     Document Type: Article

References (20)

1. Valayannopoulos V., Nicely H., Harmatz P., Turbeville S. Mucopolysaccharidosis VI. Orphanet J. Rare Dis. 2010, 5:5.
2. Neufeld E., Muenzer J. The mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease 2001, 3421-3452. McGraw-Hill, Inc., New York, NY. 8th ed. C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle (Eds.).
3. Wraith J.E. Mucopolysacharidoses type VI (Maroteaux Lamy Syndrome. Lysosomal Storage Diseases 2007, 447-456. NY, Springer, New York. J.A. Barranger, M.A. Cabrera-Salazar (Eds.).
4. Harmatz P., Whitley C.B., Waber L., et al. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J. Pediatr. 2004, 144:574-580.
5. Harmatz P., Ketteridge D., Giugliani R., et al. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48weeks in a Phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase. Pediatrics 2005, 115(6):e681-e689.
6. Harmatz P., Giugliani R., Schwartz I., et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase 3, randomized, double-blind, placebo controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J. Pediatr. 2006, 148:533-539.
7. Auclair D., Hopwood J.J., Brooks D.A., Lemontt J.F., Crawley A.C. Replacement therapy in mucopolysaccharidosis type VI: advantages of early onset of therapy. Mol. Genet. Metab. 2003, 78:163-174.
8. McGill J.J., Inwood A.C., Coman D.J., Lipke M., de Lore D., Swidler S.J., Hopwood J.J. Enzyme replacement therapy for mucopolysaccharidosis VI from 8weeks of age-a sibling control study. Clin. Genet. 2010, 77:492-498.
9. Furujoa M., Kuboa T., Kosugab M., Okuyama T. Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI. Mol. Genet. Metab. 2011, 104:597-602.
10. Magalhaes A., Teles E., Breda J., Nicely H., Turbeville S. Ophthalmologic evaluation of MPS VI patients following treatment with galsulfase enzyme replacement therapy. J. Inherit. Metab. Dis. 2007, 30(Suppl. 1):1-178. Poster presented at: Society for the Study of Inborn Errors of Metabolism(SSIEM) annual meeting; September, 2007; Hamburg Germany. Abstract available at, [abstract 466-P]. 10.1007/s10545-007-9987-1.
11. Kim K.H., Decker C., Burton B.K. Management of difficult infusion related reactions in a young patient with mucopolysaccharidosis type VI on Naglazyme therapy. Pediatrics 2008, 121:e714-e717.
12. Giugliani R., Bender F., Amorim T., et al. Newborn screening for MPS VI in a high-incidence area of northeast Brazil: preliminary results of a pilot program. Poster presented at: WORLD Symposium of the Lysosomal Disease Network; February, 2012; San Diego, California. Abstract available at. Mol. Genet. Metab. 2012, 105:S31.
13. Gallegos-Arreola M.P., Machorro-Lazo M.V., Flores-Martinez S.E., Zuniga-Gonzalez G.M., Figuera L.E., Gonzalez-Noriega A., Sanchez-Corona J. Urinary glycosaminoglycan excretion in healthy subjects and in subjects with mucopolysaccharidoses. Arch. Med. Res. 2000, 31:505-510.
14. Giugliani R., Harmatz P., Wraith J.E. Management guidelines for mucopolysaccharidosis VI. Pediatrics 2007, 120:405-418.
15. Decker C., Yu Z.F., Giugliani R., et al. Enzyme replacement therapy for mucopolysaccharidosis VI: growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. J. Pediatr. Rehabil. Med. 2010, 3(2):89-100.
16. Braunlin E.A., Berry J.M., Whitley C.B. Cardiac finding after enzyme replacement therapy for mucopolysaccharidosis type I. Am. J. Cardiol. 2006, 98:416-418.
17. Mut M., Cila A., Varli K., Akalan N. Multilevel myelopathy in Maroteaux-Lamy syndrome and review of the literature. Clin. Neurol. Neurosurg. 2005, 107:230-235.
18. Horovitz D.D.G., Magalhães T.S.C.P., Pena e Costa A., Carelli L.E., Souza e Silva P., Linhares e Riello A.P.F., Llerena J. Spinal cord compression in young children with type VI mucopolysaccharidosis. Mol. Genet. Metab. 2011, 104:295-300.
19. Karageorgos L., Brooks D.A., Pollard A., et al. Mutational analysis of 105 mucopolysaccharidosis type VI patients. Hum. Mutat. 2007, 28:897-903.
20. Azevedo A.C., Schwartz I.V., Kalakun L., et al. Clinical and biochemical study of 28 patients with mucopolysaccharidosis type VI. Clin. Genet. 2004, 66:208-213.