FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W1985193566> ?p ?o ?g. }

• W1985193566 endingPage "240" @default.
• W1985193566 startingPage "229" @default.
• W1985193566 abstract "OBJECTIVE. Our goal was to evaluate the long-term safety and efficacy of recombinant human α-l-iduronidase (laronidase) in patients with mucopolysaccharidosis I. PATIENTS AND METHODS. All 45 patients who completed a 26-week, double-blind, placebo-controlled trial of laronidase were enrolled in a 3.5-year open-label extension study. Mean patient age at baseline was 16 (range: 6–43) years. All patients had attenuated disease (84% Hurler-Scheie, 16% Scheie phenotypes). Clinical, biochemical, and health outcomes measures were evaluated through the extension phase. Changes are presented as the mean ± SEM. RESULTS. All 40 patients (89%) who completed the trial received at least 80% of scheduled infusions. As shown in earlier trials, urinary glycosaminoglycan levels decreased within the first 12 weeks and liver volume decreased within the first year. Percent predicted forced vital capacity remained stable, with a linear slope of −0.78 percentage points per year. The 6-minute walk distance increased 31.7 ± 10.2 m in the first 2 years, with a final gain of 17.1 ± 16.8 m. Improvements in the apnea/hypopnea index (decrease of 7.6 ± 4.5 events per hour among the patients with significant baseline sleep apnea) and shoulder flexion (increase of 17.4° ± 3.6°) were most rapid during the first 2 years. Improvements in the Child Health Assessment Questionnaire/Health Assessment Questionnaire disability index (decrease of 0.31 ± 0.11, signifying a clinically meaningful improvement in activities of daily living) were gradual and sustained over the treatment period. Laronidase infusions were generally well tolerated except in 1 patient who experienced an anaphylactic reaction. Infusion-associated reactions, which occurred in 53% of the patients, were mostly mild, easily managed, and decreased markedly after 6 months. One patient died as a result of an upper respiratory infection unrelated to treatment. Antibodies to laronidase developed in 93% of the patients; 29% of the patients were seronegative at their last assessment. CONCLUSIONS. This trial demonstrates the long-term clinical benefit and safety of laronidase in attenuated patients with mucopolysaccharidosis I and highlights the magnitude and chronology of treatment effects. Prompt diagnosis and early treatment will maximize treatment outcomes." @default.
• W1985193566 created "2016-06-24" @default.
• W1985193566 creator A5003149248 @default.
• W1985193566 creator A5006551650 @default.
• W1985193566 creator A5014057972 @default.
• W1985193566 creator A5015169555 @default.
• W1985193566 creator A5017093187 @default.
• W1985193566 creator A5035003505 @default.
• W1985193566 creator A5049974643 @default.
• W1985193566 creator A5072415019 @default.
• W1985193566 creator A5080341479 @default.
• W1985193566 creator A5084371637 @default.
• W1985193566 creator A5085315279 @default.
• W1985193566 date "2009-01-01" @default.
• W1985193566 modified "2023-10-16" @default.
• W1985193566 title "Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I" @default.
• W1985193566 cites W1990211122 @default.
• W1985193566 cites W1995086395 @default.
• W1985193566 cites W2023598701 @default.
• W1985193566 cites W2036548803 @default.
• W1985193566 cites W2041499523 @default.
• W1985193566 cites W2059192486 @default.
• W1985193566 cites W2059934840 @default.
• W1985193566 cites W2061347348 @default.
• W1985193566 cites W2077868231 @default.
• W1985193566 cites W2078809930 @default.
• W1985193566 cites W2085884984 @default.
• W1985193566 cites W2088789656 @default.
• W1985193566 cites W2093722439 @default.
• W1985193566 cites W2112165124 @default.
• W1985193566 cites W2136255603 @default.
• W1985193566 cites W2140044661 @default.
• W1985193566 cites W2140806188 @default.
• W1985193566 cites W2312413018 @default.
• W1985193566 cites W2482445792 @default.
• W1985193566 cites W4249200669 @default.
• W1985193566 cites W4313194205 @default.
• W1985193566 doi "https://doi.org/10.1542/peds.2007-3847" @default.
• W1985193566 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/19117887" @default.
• W1985193566 hasPublicationYear "2009" @default.
• W1985193566 type Work @default.
• W1985193566 sameAs 1985193566 @default.
• W1985193566 citedByCount "293" @default.
• W1985193566 countsByYear W19851935662012 @default.
• W1985193566 countsByYear W19851935662013 @default.
• W1985193566 countsByYear W19851935662014 @default.
• W1985193566 countsByYear W19851935662015 @default.
• W1985193566 countsByYear W19851935662016 @default.
• W1985193566 countsByYear W19851935662017 @default.
• W1985193566 countsByYear W19851935662018 @default.
• W1985193566 countsByYear W19851935662019 @default.
• W1985193566 countsByYear W19851935662020 @default.
• W1985193566 countsByYear W19851935662021 @default.
• W1985193566 countsByYear W19851935662022 @default.
• W1985193566 countsByYear W19851935662023 @default.
• W1985193566 crossrefType "journal-article" @default.
• W1985193566 hasAuthorship W1985193566A5003149248 @default.
• W1985193566 hasAuthorship W1985193566A5006551650 @default.
• W1985193566 hasAuthorship W1985193566A5014057972 @default.
• W1985193566 hasAuthorship W1985193566A5015169555 @default.
• W1985193566 hasAuthorship W1985193566A5017093187 @default.
• W1985193566 hasAuthorship W1985193566A5035003505 @default.
• W1985193566 hasAuthorship W1985193566A5049974643 @default.
• W1985193566 hasAuthorship W1985193566A5072415019 @default.
• W1985193566 hasAuthorship W1985193566A5080341479 @default.
• W1985193566 hasAuthorship W1985193566A5084371637 @default.
• W1985193566 hasAuthorship W1985193566A5085315279 @default.
• W1985193566 hasConcept C126322002 @default.
• W1985193566 hasConcept C141071460 @default.
• W1985193566 hasConcept C142724271 @default.
• W1985193566 hasConcept C1862650 @default.
• W1985193566 hasConcept C187212893 @default.
• W1985193566 hasConcept C197934379 @default.
• W1985193566 hasConcept C204787440 @default.
• W1985193566 hasConcept C27081682 @default.
• W1985193566 hasConcept C2776890885 @default.
• W1985193566 hasConcept C535046627 @default.
• W1985193566 hasConcept C71924100 @default.
• W1985193566 hasConcept C77411442 @default.
• W1985193566 hasConceptScore W1985193566C126322002 @default.
• W1985193566 hasConceptScore W1985193566C141071460 @default.
• W1985193566 hasConceptScore W1985193566C142724271 @default.
• W1985193566 hasConceptScore W1985193566C1862650 @default.
• W1985193566 hasConceptScore W1985193566C187212893 @default.
• W1985193566 hasConceptScore W1985193566C197934379 @default.
• W1985193566 hasConceptScore W1985193566C204787440 @default.
• W1985193566 hasConceptScore W1985193566C27081682 @default.
• W1985193566 hasConceptScore W1985193566C2776890885 @default.
• W1985193566 hasConceptScore W1985193566C535046627 @default.
• W1985193566 hasConceptScore W1985193566C71924100 @default.
• W1985193566 hasConceptScore W1985193566C77411442 @default.
• W1985193566 hasIssue "1" @default.
• W1985193566 hasLocation W19851935661 @default.
• W1985193566 hasLocation W19851935662 @default.
• W1985193566 hasOpenAccess W1985193566 @default.
• W1985193566 hasPrimaryLocation W19851935661 @default.
• W1985193566 hasRelatedWork W2030308013 @default.
• W1985193566 hasRelatedWork W2041904666 @default.

• next