ArticleJIMD reports2015
Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome).
Article in JIMD reports, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
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Who cites it
23 citing papers in PubMed, 37 citations in OpenAlex.
- Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.Pediatric research · 2015Trial
- Disease-specific growth charts capture characteristic growth patterns in children with PMM2 - CDG.Molecular genetics and metabolism · 2026Article
- Multisystem Involvement in a Pediatric Patient With Suspected Mucopolysaccharidosis: A Case Report.Cureus · 2024Article
- Standardized growth charts for children with osteogenesis imperfecta.Pediatric research · 2023Article
- Growth patterns in patients with mucopolysaccharidosis VII.Molecular genetics and metabolism reports · 2023Article
- Functioning profiles of individuals with Mucopolysaccharidosis according to the International Classification of Functioning.European journal of physical and rehabilitation medicine · 2022Article
- Mucopolysaccharidosis Type VI, an Updated Overview of the Disease.International journal of molecular sciences · 2021Review
- Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series.Orphanet journal of rare diseases · 2021Article
- Disease burden, management patterns and multidisciplinary clinical approaches for patients with MPS IVA and VI in selected Latin American Countries.Molecular genetics and metabolism reports · 2021Article
- "Growth patterns in children with mucopolysaccharidosis type I-Hurler after hematopoietic stem cell transplantation: Comparison with untreated patients".Molecular genetics and metabolism reports · 2021Article
- Failures of Endochondral Ossification in the Mucopolysaccharidoses.Current osteoporosis reports · 2020Review
- Mowat-Wilson syndrome: growth charts.Orphanet journal of rare diseases · 2020Article
- Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.International journal of molecular sciences · 2020Review
- Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry.American journal of medical genetics. Part A · 2019Article
- Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future.Journal of human genetics · 2019Review
- Relationships among Height, Weight, Body Mass Index, and Age in Taiwanese Children with Different Types of Mucopolysaccharidoses.Diagnostics (Basel, Switzerland) · 2019Article
- The use of recombinant human growth hormone in patients with Mucopolysaccharidoses and growth hormone deficiency: a case series.Italian journal of pediatrics · 2019Article
- Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III).Orphanet journal of rare diseases · 2019Article
- Growth impairment and limited range of joint motion in children should raise suspicion of an attenuated form of mucopolysaccharidosis: expert opinion.European journal of pediatrics · 2019Article
- Dental-craniofacial manifestation and treatment of rare diseases.International journal of oral science · 2019Review
Corrections and comments
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Authors and funding
6 authors at 4 institutions in 3 countries.
Funding
Abstract
backgroundThe skeletal phenotype of mucopolysaccharidosis VI (MPS VI) is characterized by short stature and growth failure.
objectiveThe purpose of this study was to construct reference growth curves for MPS VI patients with rapidly and slowly progressive disease.
methodsWe pooled cross-sectional and longitudinal height for age data from galsulfase (Naglazyme(®), BioMarin Pharmaceutical Inc.), treatment naïve patients (n = 269) who participated in various MPS VI studies, including galsulfase clinical trials and their extension programs, the MPS VI clinical surveillance program (CSP), and the MPS VI survey and resurvey studies, to construct growth charts for the MPS VI population. There were 229 patients included in this study, of which data from 207 patients ≤25 years of age with 513 height measurements were used for constructing reference growth curves.
resultsHeight for age growth curves for the 5th, 10th, 25th, 50th, 75th, 90th, and 95th percentiles were constructed for patients with rapidly and slowly progressing disease defined by the pre-enzyme replacement therapy (ERT) uGAG levels of > or ≤200 μg/mg creatinine. The mean (SD) pre-ERT uGAG levels were 481.0 (218.6) and 97.8 (56.3) μg/mg creatinine for the patients ≤25 years of age with rapidly (n = 131) and slowly (n = 76) progressing MPS VI disease, respectively. The median growth curves for patients with ≤ and >200 μg/mg creatinine were above and below the median (50th percentile) growth curve for the entire MPS VI population.
conclusionMPS VI growth charts have been developed to assist in the clinical management of MPS VI patients.
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.