ReviewDrug design, development and therapy2012
Imiglucerase in the treatment of Gaucher disease: a history and perspective.
Review in Drug design, development and therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 76 citations in OpenAlex.
- Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results.American journal of hematology · 2021Trial
- Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.American journal of hematology · 2016Trial
- Glycoprotein non-metastatic melanoma protein B is a biomarker of inflammation in individuals with Gaucher disease: relationship to clinico-pathological subtypes.Orphanet journal of rare diseases · 2025Article
- FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- A 20-Year Longitudinal Study of Plasma Chitotriosidase Activity in Treated Gaucher Disease Type 1 and 3 Patients-A Qualitative and Quantitative Approach.Biomolecules · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Natural history and management of liver dysfunction in lysosomal storage disorders.World journal of hepatology · 2022Review
- Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy.Journal of clinical medicine · 2022Article
- Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise.Orphanet journal of rare diseases · 2022Review
- Strategies for Glycoengineering Therapeutic Proteins.Frontiers in chemistry · 2022Review
- In-depth phenotyping for clinical stratification of Gaucher disease.Orphanet journal of rare diseases · 2021Article
- Theoretical Analysis of the Built-in Metabolic Pathway Effect on the Metabolism of Erythrocyte-Bioreactors That Neutralize Ammonium.Metabolites · 2021Article
- Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Nature communications · 2020Article
- A baculoviral system for the production of human β-glucocerebrosidase enables atomic resolution analysis.Acta crystallographica. Section D, Structural biology · 2020Article
- A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease.Orphanet journal of rare diseases · 2018Review
- Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options.Hospital pharmacy · 2016Article
- Gaucher disease. Unusual presentation and mini-review.Neurosciences (Riyadh, Saudi Arabia) · 2015Review
- Tool compounds robustly increase turnover of an artificial substrate by glucocerebrosidase in human brain lysates.PloS one · 2015Article
- The impracticality of biomedical rejuvenation therapies: translational and pharmacological barriers.Rejuvenation research · 2014Article
- Pathogen safety of long-term treatments for bleeding disorders: still relevant to current practice.Haematologica · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The scientific and therapeutic development of imiglucerase (Cerezyme(®)) by the Genzyme Corporation is a paradigm case for a critical examination of current trends in biotechnology. In this article the authors argue that contemporary interest in treatments for rare diseases by major pharmaceutical companies stems in large part from an exception among rarities: the astonishing commercial success of Cerezyme. The fortunes of the Genzyme Corporation, latterly acquired by global giant Sanofi SA, were founded on the evolution of a blockbuster therapy for a single but, as it turns out, propitious ultra-orphan disorder: Gaucher disease.
Indexed as
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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.