ArticleJournal of inherited metabolic disease2010
Enzyme replacement therapy for mucopolysaccharidosis VI--experience in Taiwan.
Article in Journal of inherited metabolic disease, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03017677 (The Establishment of a Cross-specialty Collaboration Platform Among Different Medical Specialties Based on High-risk Criteria for Mucopolysaccharidosis Confirmative Diagnosis), which is not on this map. Cited by 11 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
The Establishment of a Cross-specialty Collaboration Platform Among Different Medical Specialties Based on High-risk Criteria for Mucopolysaccharidosis Confirmative Diagnosis
Who cites it
11 citing papers in PubMed, 26 citations in OpenAlex.
- Epidemiology of Mucopolysaccharidoses Update.Diagnostics (Basel, Switzerland) · 2021Review
- Home treatment of type VI mucopolysaccharidosis (Maroteaux-Lamy syndrome) an alternative at this time of COVID-19 pandemic: A case in Peru.Clinical case reports · 2020Article
- An At-Risk Population Screening Program for Mucopolysaccharidoses by Measuring Urinary Glycosaminoglycans in Taiwan.Diagnostics (Basel, Switzerland) · 2019Article
- The relationships between urinary glycosaminoglycan levels and phenotypes of mucopolysaccharidoses.Molecular genetics & genomic medicine · 2018Article
- Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues.Orphanet journal of rare diseases · 2016Review
- Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series.Molecular genetics and metabolism reports · 2016Article
- A modified liquid chromatography/tandem mass spectrometry method for predominant disaccharide units of urinary glycosaminoglycans in patients with mucopolysaccharidoses.Orphanet journal of rare diseases · 2014Article
- Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study.American journal of medical genetics. Part A · 2014Article
- Respiratory manifestations in patients with inherited metabolic diseases.European respiratory review : an official journal of the European Respiratory Society · 2013Review
- Assessment of bone mineral density by dual energy x-ray absorptiometry in patients with mucopolysaccharidoses.Orphanet journal of rare diseases · 2013Article
- Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 8 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Information regarding the clinical outcome of enzyme replacement therapy (ERT) with recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for mucopolysaccharidosis (MPS) VI in Asian patients is limited. We reviewed nine Taiwanese patients with MPS VI (four males and five females; age range 1.4-21.1 years) treated with weekly intravenous infusions of rhASB (1.0 mg/kg) for at least 2 years. We assessed the biochemical and clinical response every 3 months. After 2 years of treatment, seven patients experienced improvement over baseline in the 6-min walk by a mean of 69.3 m (27.3%), and seven also increased the 3-min stair climb by a mean of 47 steps (35.7%). Shoulder range of motion in all patients improved, and Joint Pain and Stiffness Questionnaire scores improved by 0.597 points (30.5%). Four patients had improved pulmonary function [forced expiratory volume in 1 s increased by 0.130 L (26.3%) and forced vital capacity by 0.148 L (27.6%)]. The respiratory disturbance index decreased in the four patients who underwent polysomnography. A mean overall 51% decrease in urinary glycosaminoglycan excretion indicated a satisfactory biochemical response. ERT was well tolerated by all patients. This treatment is thus beneficial and appears to be safe for treatment of MPS VI in Taiwanese patients.
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