Results: 111
Total synthesis of N -butyl-1-deoxynojirimycin.
- Published in:
- Journal of Carbohydrate Chemistry, 2016, v. 35, n. 8/9, p. 445, doi. 10.1080/07328303.2017.1330415
- By:
- Publication type:
- Article
Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.
- Published in:
- Molecular Genetics & Genomic Medicine, 2018, v. 6, n. 1, p. 27, doi. 10.1002/mgg3.339
- By:
- Publication type:
- Article
FROM SYMPTOMS AND SIGNS TO DIAGNOSIS IN A RARE DISEASE, TYPE I GAUCHER DISEASE.
- Published in:
- 2018
- By:
- Publication type:
- Case Study
Evaluation of the Nutritional Status of Gaucher Disease Type I Patients under Enzyme Replacement Treatment.
- Published in:
- Nutrients, 2022, v. 14, n. 15, p. 3180, doi. 10.3390/nu14153180
- By:
- Publication type:
- Article
IMMUNOGLOBULIN HEAVY CHAIN GENE REARRANGEMENTS IN PATIENTS WITH GAUCHER DISEASE.
- Published in:
- Journal of Medical Biochemistry, 2018, v. 37, n. 3, p. 307, doi. 10.1515/jomb-2017-0061
- By:
- Publication type:
- Article
Low-dose high-frequency enzyme replacement therapy for very young children with severe Gaucher...
- Published in:
- 1994
- By:
- Publication type:
- Abstract
From the National Institutes of Health.
- Published in:
- 1991
- By:
- Publication type:
- journal article
Pulmonary hypertension developing after alglucerase therapy in two patients with type 1 Gaucher..
- Published in:
- Annals of Internal Medicine, 1996, v. 125, n. 11, p. 901, doi. 10.7326/0003-4819-125-11-199612010-00005
- By:
- Publication type:
- Article
Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated...
- Published in:
- Annals of Internal Medicine, 1995, v. 122, n. 1, p. 33, doi. 10.7326/0003-4819-122-1-199501010-00005
- By:
- Publication type:
- Article
Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.
- Published in:
- Orphanet Journal of Rare Diseases, 2014, v. 9, n. 1, p. 2, doi. 10.1186/1750-1172-9-51
- By:
- Publication type:
- Article
Reducing selection bias in case-control studies from rare disease registries.
- Published in:
- 2011
- By:
- Publication type:
- journal article
Reply to vom dahl et al.
- Published in:
- British Journal of Haematology, 2001, v. 113, n. 4, p. 1086, doi. 10.1046/j.1365-2141.2001.02821-10.x
- By:
- Publication type:
- Article
Interruption in enzyme replacement therapy for gaucher disease.
- Published in:
- British Journal of Haematology, 2001, v. 113, n. 4, p. 1087, doi. 10.1046/j.1365-2141.2001.02821-11.x
- By:
- Publication type:
- Article
Withdrawal of enzyme replacement therapy in Gaucher's disease.
- Published in:
- British Journal of Haematology, 2000, v. 110, n. 2, p. 488, doi. 10.1046/j.1365-2141.2000.02177.x
- By:
- Publication type:
- Article
Discussed Poster Abstracts.
- Published in:
- 2018
- Publication type:
- Abstract
Recombinant Glucocerebrosidase (Imiglucerase) as a Therapy for Gaucher Disease.
- Published in:
- BioDrugs, 2010, v. 24, n. 1, p. 41, doi. 10.2165/11318540-000000000-00000
- By:
- Publication type:
- Article
Alglucerase: Practical Guidance on Appropriate Dosage and Administration in Patients with Gaucher Disease.
- Published in:
- BioDrugs, 1998, v. 9, n. 1, p. 11
- By:
- Publication type:
- Article
Cytokines in Gaucher disease: Role in the pathogenesis of bone and pulmonary disease.
- Published in:
- Egyptian Journal of Medical Human Genetics, 2015, v. 16, n. 3, p. 207, doi. 10.1016/j.ejmhg.2015.02.001
- By:
- Publication type:
- Article
Structure of acid β-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
- Published in:
- 2007
- By:
- Publication type:
- Letter
Gene variants of osteoprotegerin, estrogen-, calcitonin- and vitamin D-receptor genes and serum markers of bone metabolism in patients with Gaucher disease type 1.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Profile of eliglustat tartrate in the management of Gaucher disease.
- Published in:
- 2016
- By:
- Publication type:
- journal article
Homozygosity for two mild glucocerebrosidase mutations of probable Iberian origin.
- Published in:
- Clinical Genetics, 1999, v. 56, n. 1, p. 100, doi. 10.1034/j.1399-0004.1999.560117.x
- By:
- Publication type:
- Article
Glycosylation and functionality of recombinant ß-glucocerebrosidase from various production systems.
- Published in:
- Bioscience Reports, 2013, v. 33, n. 5, p. 771, doi. 10.1042/BSR20130081
- By:
- Publication type:
- Article
Gaucher's disease oral therapy gets nod from FDA.
- Published in:
- Nature Biotechnology, 2014, v. 32, n. 10, p. 970, doi. 10.1038/nbt1014-970
- By:
- Publication type:
- Article
First plant-made biologic approved.
- Published in:
- Nature Biotechnology, 2012, v. 30, n. 6, p. 472, doi. 10.1038/nbt0612-472
- By:
- Publication type:
- Article
Shire's replacement enzymes validate gene activation.
- Published in:
- Nature Biotechnology, 2010, v. 28, n. 11, p. 1139, doi. 10.1038/nbt1110-1139a
- By:
- Publication type:
- Article
Pfizer stakes a claim in plant cell–made biopharmaceuticals.
- Published in:
- Nature Biotechnology, 2010, v. 28, n. 2, p. 107, doi. 10.1038/nbt0210-107
- By:
- Publication type:
- Article
Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment.
- Published in:
- Nature Biotechnology, 2008, v. 26, n. 8, p. 901, doi. 10.1038/nbt.1484
- By:
- Publication type:
- Article
RIPK3 as a potential therapeutic target for Gaucher's disease.
- Published in:
- Nature Medicine, 2014, v. 20, n. 2, p. 204, doi. 10.1038/nm.3449
- By:
- Publication type:
- Article
Abundant Hepatic Gaucher-Like Cells Following Chemotherapy and Bone Marrow Transplantation for Hematologic Malignancy: Report of Two Cases.
- Published in:
- International Journal of Surgical Pathology, 2013, v. 21, n. 1, p. 89, doi. 10.1177/1066896912456081
- By:
- Publication type:
- Article
2012 The year in review.
- Published in:
- Chemistry & Industry, 2012, v. 76, n. 11, p. 32
- By:
- Publication type:
- Article
Lysosomal storage diseases market.
- Published in:
- Nature Reviews Drug Discovery, 2004, v. 3, n. 1, p. 9, doi. 10.1038/nrd1286
- By:
- Publication type:
- Article
β-Glycosphingolipids as Mediators of Both Inflammation and Immune Tolerance: A Manifestation of Randomness in Biological Systems.
- Published in:
- Frontiers in Immunology, 2019, p. N.PAG, doi. 10.3389/fimmu.2019.01143
- By:
- Publication type:
- Article
Glucocerebrosidase level in the cerebrospinal fluid during enzyme replacement therapy – unsuccessful treatment of the neurological abnormality in type 2 Gaucher disease.
- Published in:
- European Journal of Pediatrics, 2003, v. 162, n. 7/8, p. 524, doi. 10.1007/s00431-001-0859-7
- By:
- Publication type:
- Article
Improvement of neurological symptoms by enzyme replacement therapy for Gaucher disease type IIIb.
- Published in:
- 2001
- By:
- Publication type:
- Case Study
Gaucher's Disease: Diagnosis and Treatment.
- Published in:
- American Family Physician, 1994, v. 49, n. 5, p. 1231
- Publication type:
- Article
Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report.
- Published in:
- 2017
- By:
- Publication type:
- journal article
Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.
- Published in:
- 2016
- By:
- Publication type:
- Case Study
Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease.
- Published in:
- PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0128986
- By:
- Publication type:
- Article
Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model.
- Published in:
- PLoS ONE, 2010, v. 5, n. 5, p. 1, doi. 10.1371/journal.pone.0010750
- By:
- Publication type:
- Article
PATIENTS WITH GAUCHER DISEASE: RESULTS OF LONG FOLLOW UP.
- Published in:
- Erciyes Medical Journal / Erciyes Tip Dergisi, 2018, v. 40, n. 1, p. S23
- By:
- Publication type:
- Article
Echocardiographic assessment in children with Gaucher disease receiving enzyme replacement therapy.
- Published in:
- 2012
- By:
- Publication type:
- Letter
Gaucher disease in Iraqi children (Clinical, diagnostic & therapeutic aspects).
- Published in:
- Pakistan Journal of Medical Sciences, 2016, v. 32, n. 2, p. 319, doi. 10.12669/pjms.322.9316
- By:
- Publication type:
- Article
Probing the Inhibitor versus Chaperone Properties of sp2-Iminosugars towards Human β-Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease.
- Published in:
- Molecules, 2018, v. 23, n. 4, p. 927, doi. 10.3390/molecules23040927
- By:
- Publication type:
- Article
The natural history of Gaucher disease type 1 in 31 patients over a median of 15 years: a retrospective study.
- Published in:
- Internal Medicine Journal, 2024, v. 54, n. 10, p. 1661, doi. 10.1111/imj.16503
- By:
- Publication type:
- Article
Critical appraisal of observational studies on enzyme replacement therapy for Gaucher's Disease.
- Published in:
- JBES: Brazilian Journal of Health Economics / Jornal Brasileiro de Economia da Saúde, 2015, v. 7, n. 3, p. 148
- By:
- Publication type:
- Article
Treatment of severe pulmonary hypertension with inhaled iloprost.
- Published in:
- 2003
- By:
- Publication type:
- Case Study
Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.
- Published in:
- 2018
- By:
- Publication type:
- journal article
A multicentre observational study for early diagnosis of Gaucher disease in patients with Splenomegaly and/or Thrombocytopenia.
- Published in:
- European Journal of Haematology, 2016, v. 96, n. 4, p. 352, doi. 10.1111/ejh.12596
- By:
- Publication type:
- Article
Therapeutic potential of resveratrol for the treatment of type III Gaucher disease.
- Published in:
- Neurology Asia, 2015, v. 20, n. 1, p. 43
- By:
- Publication type:
- Article