Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome is a research paper published in Journal of Inherited Metabolic Disease (2006). On theSindex it has a DataRank of 2.1. It has been cited 23 times, with 23 citing works in its 1-hop citation network.
SummaryEnzyme replacement therapy (ERT) with laronidase, recombinant α‐l‐iduronidase, for mucopolysaccharidosis type I (MPS I) has been clinically available since April 2003. Pre‐approval studies were performed on patients with the more attenuated forms of MPS I, Hurler–Scheie and Scheie syndromes. The clinical efficacy of laronidase on the severe form of MPS I, Hurler syndrome, is not well known. We present a patient with Hurler syndrome who has been treated with laronidase for 3 years. Clinically, the patient demonstrated improvement in urinary glycosaminoglycan (GAG) levels and hepatomegaly, but continued to experience decline in respiratory status, musculoskeletal and spinal involvement, and developmental skills. Overall, the benefit of ERT with laronidase in advanced Hurler syndrome appeared to be minimal in this patient.
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Base Score Contribution
0.477
From this paper's citation signal
Citation Network Contribution
1.6
From 21 citing papers with measurable signal
DataRank blends this paper's own citation count with the influence of the papers that cite it. Here, roughly 23% comes from its base citations and 77% from the citation network (21 citing papers contributed measurable signal).
Citers are pulled from OpenAlex sorted by cited_by_count:descand capped per paper, so when the cap binds we keep the highest-signal references and the score is reproducible across reruns.
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