ArticleExpert opinion on orphan drugs2013
Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).
Article in Expert opinion on orphan drugs, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
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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.
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Who cites it
17 citing papers in PubMed, 29 citations in OpenAlex.
- Evaluation of Gait Pattern and Lower Extremity Kinematics of Children with Morquio Syndrome (MPS IV).Diagnostics (Basel, Switzerland) · 2021Article
- Failures of Endochondral Ossification in the Mucopolysaccharidoses.Current osteoporosis reports · 2020Review
- Modeling Morquio A Syndrome: An Anthropometric Study of Body Characteristics and Stature.Diagnostics (Basel, Switzerland) · 2020Article
- Newborn screening for Morquio disease and other lysosomal storage diseases: results from the 8-plex assay for 70,000 newborns.Orphanet journal of rare diseases · 2020Article
- Pediatric Dental Management of an Uncommon Case of Mucopolysaccharidosis Type IV A (Morquio A Syndrome): A Case Report of a Three-Year Follow-Up.Case reports in dentistry · 2020Article
- Normalization of glycosaminoglycan-derived disaccharides detected by tandem mass spectrometry assay for the diagnosis of mucopolysaccharidosis.Scientific reports · 2019Article
- Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan.Orphanet journal of rare diseases · 2018Article
- Bio-Plex immunoassay measuring the quantity of lysosomalBMJ open · 2017Article
- Skeletal Dysplasias: Growing Therapy for Growing Bones.Frontiers in pharmacology · 2017Review
- Hematopoietic stem cell transplantation for Morquio A syndrome.Molecular genetics and metabolism · 2016Article
- Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations.Expert opinion on orphan drugs · 2015Article
- Non-invasive pulmonary function test on Morquio patients.Molecular genetics and metabolism · 2015Article
- Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?Molecular genetics and metabolism · 2015Article
- Therapies for the bone in mucopolysaccharidoses.Molecular genetics and metabolism · 2015Review
- Di-sulfated Keratan Sulfate as a Novel Biomarker for Mucopolysaccharidosis II, IVA, and IVB.JIMD reports · 2015Article
- Chondroitin 6-Sulfate as a Novel Biomarker for Mucopolysaccharidosis IVA and VII.JIMD reports · 2014Article
- Mucopolysaccharidosis IVA and glycosaminoglycans.Molecular genetics and metabolismReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 5 institutions in 4 countries.
Funding
Abstract
introductionMorquio A syndrome (mucopolysaccharidosis type IVA, MPS IVA) is one of the lysosomal storage diseases and is caused by the deficiency of AREAS COVERED: This article describes the advanced therapies of Morquio A, focused on enzyme replacement therapy (ERT) and gene therapy to deliver the drug to avascular bone lesions. ERT and gene therapies for other types of MPS are also discussed, which provide therapeutic efficacy to bone lesions. EXPERT OPINION: ERT, gene therapy and hematopietic stem therapy are clinically and/or experimentally conducted. However, there is no effective curative therapy for bone lesion to date. One of the limitations for Morquio A therapy is that targeting avascular cartilage tissues remains an unmet challenge. ERT or gene therapy with bone-targeting system will improve the bone pathology and skeletal manifestations more efficiently.
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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.