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Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.
J Child Neurol 2007; 22(5):565-73JC

Abstract

Pompe disease is a rare autosomal recessive myopathy due to the deficiency of lysosomal acid alpha-glucosidase. Clinical phenotypes range from the severe classic infantile form (hypotonia and hypertrophic cardiomyopathy), to milder late onset forms (skeletal myopathy and absence of significant heart involvement). Enzyme replacement therapy with recombinant human alpha-glucosidase derived from either rabbit milk or Chinese hamster ovary cells has been introduced and is undergoing clinical trials. Reported is a long-term follow-up of 3 Pompe patients presenting without cardiomyopathy, treated with recombinant human alpha-glucosidase derived from Chinese hamster ovary cells. This study suggests that enzyme replacement therapy can lead to significant motor and respiratory improvement in the subgroup of patients who start the therapy before extensive muscle damage has occurred. The recombinant enzyme derived from Chinese hamster ovary cells, administered at doses significantly higher than previously reported, appears to have the same safety as the drug derived from rabbit milk.

Authors+Show Affiliations

Department of Pediatrics, Federico II University, Naples, Italy.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

17690063

Citation

Rossi, Massimiliano, et al. "Long-term Enzyme Replacement Therapy for Pompe Disease With Recombinant Human Alpha-glucosidase Derived From Chinese Hamster Ovary Cells." Journal of Child Neurology, vol. 22, no. 5, 2007, pp. 565-73.
Rossi M, Parenti G, Della Casa R, et al. Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells. J Child Neurol. 2007;22(5):565-73.
Rossi, M., Parenti, G., Della Casa, R., Romano, A., Mansi, G., Agovino, T., ... Andria, G. (2007). Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells. Journal of Child Neurology, 22(5), pp. 565-73.
Rossi M, et al. Long-term Enzyme Replacement Therapy for Pompe Disease With Recombinant Human Alpha-glucosidase Derived From Chinese Hamster Ovary Cells. J Child Neurol. 2007;22(5):565-73. PubMed PMID: 17690063.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells. AU - Rossi,Massimiliano, AU - Parenti,Giancarlo, AU - Della Casa,Roberto, AU - Romano,Alfonso, AU - Mansi,Giuseppina, AU - Agovino,Teresa, AU - Rosapepe,Felice, AU - Vosa,Carlo, AU - Del Giudice,Ennio, AU - Andria,Generoso, PY - 2007/8/11/pubmed PY - 2007/10/3/medline PY - 2007/8/11/entrez SP - 565 EP - 73 JF - Journal of child neurology JO - J. Child Neurol. VL - 22 IS - 5 N2 - Pompe disease is a rare autosomal recessive myopathy due to the deficiency of lysosomal acid alpha-glucosidase. Clinical phenotypes range from the severe classic infantile form (hypotonia and hypertrophic cardiomyopathy), to milder late onset forms (skeletal myopathy and absence of significant heart involvement). Enzyme replacement therapy with recombinant human alpha-glucosidase derived from either rabbit milk or Chinese hamster ovary cells has been introduced and is undergoing clinical trials. Reported is a long-term follow-up of 3 Pompe patients presenting without cardiomyopathy, treated with recombinant human alpha-glucosidase derived from Chinese hamster ovary cells. This study suggests that enzyme replacement therapy can lead to significant motor and respiratory improvement in the subgroup of patients who start the therapy before extensive muscle damage has occurred. The recombinant enzyme derived from Chinese hamster ovary cells, administered at doses significantly higher than previously reported, appears to have the same safety as the drug derived from rabbit milk. SN - 0883-0738 UR - https://www.unboundmedicine.com/medline/citation/17690063/Long_term_enzyme_replacement_therapy_for_pompe_disease_with_recombinant_human_alpha_glucosidase_derived_from_chinese_hamster_ovary_cells_ L2 - http://journals.sagepub.com/doi/full/10.1177/0883073807302598?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -