ArticleOrphanet journal of rare diseases2014
Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
Article in Orphanet journal of rare diseases, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 42 citations in OpenAlex.
- Gaucher disease type 3c: Expanding the clinical spectrum of an ultra-rare disease.JIMD reports · 2024Article
- A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study.Journal of clinical medicine · 2024Article
- Article
- Economic burden and health related quality of life of ultra-rare Gaucher disease in China.Orphanet journal of rare diseases · 2021Article
- Cardiac Manifestations in a Group of Romanian Patients with Gaucher Disease Type 1 (a Monocentric Study).Diagnostics (Basel, Switzerland) · 2021Article
- Sphingolipid lysosomal storage diseases: from bench to bedside.Lipids in health and disease · 2021Review
- A Cross-Sectional Retrospective Study of Non-Splenectomized and Never-Treated Patients with Type 1 Gaucher Disease.Journal of clinical medicine · 2020Article
- Effect of Substrate Reduction Therapy in Comparison to Enzyme Replacement Therapy on Immune Aspects and Bone Involvement in Gaucher Disease.Biomolecules · 2020Article
- Epidemiology, Pathogenesis, and Clinical Approach in Group 5 Pulmonary Hypertension.Frontiers in medicine · 2020Review
- Subsidizing PGD: The Moral Case for Funding Genetic Selection.Journal of bioethical inquiry · 2019Article
- Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.Molecular genetics & genomic medicine · 2018Article
- Medical Management of Pulmonary Hypertension with Unclear and/or Multifactorial Mechanisms (Group 5): Is There a Role for Pulmonary Arterial Hypertension Medications?Current hypertension reports · 2017Review
- Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease.Orphanet journal of rare diseases · 2017Article
- A Novel Functional Missense Mutation p.T219A in Type 1 Gaucher's Disease.Chinese medical journal · 2016Article
- Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.Orphanet journal of rare diseases · 2016Article
- Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3).Orphanet journal of rare diseases · 2015Article
- Gaucher disease: the role of the specialist on metabolic bone diseases.Clinical cases in mineral and bone metabolism : the official journal of the Italian Society of Osteoporosis, Mineral Metabolism, and Skeletal DiseasesReview
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Long-term complications and associated conditions of type 1 Gaucher Disease (GD) can include splenectomy, bone complications, pulmonary hypertension, Parkinson disease and malignancies. Enzyme replacement therapy (ERT) reverses cytopenia and reduces organomegaly. To study the effects of ERT on long-term complications and associated conditions, the course of Gaucher disease was modelled.
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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.