Neal Weinreb

Summary

Publications

  1. ncbi request reprint Imiglucerase (Cerezyme) improves quality of life in patients with skeletal manifestations of Gaucher disease
    N Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Coral Springs, FL 33065, USA
    Clin Genet 71:576-88. 2007
  2. doi request reprint A validated disease severity scoring system for adults with type 1 Gaucher disease
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Disorders, Coral Springs, FL 33065, USA
    Genet Med 12:44-51. 2010
  3. ncbi request reprint Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes
    Marissa Orenstein
    Dr John T, Macdonald Foundation Department of Human Genetics, Miller School of Medicine, Miami, FL, USA
    Orphanet J Rare Dis 9:45. 2014
  4. ncbi request reprint Oral small molecule therapy for lysosomal storage diseases
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Boca Raton, Florida 33433, USA
    Pediatr Endocrinol Rev 11:77-90. 2013
  5. pmc Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Inc, Northwest Oncology Hematology Associates PA, Coral Springs, FL 33065, USA
    J Inherit Metab Dis 36:543-53. 2013
  6. ncbi request reprint Causes of death due to hematological and non-hematological cancers in 57 US patients with type 1 Gaucher Disease who were never treated with enzyme replacement therapy
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Boca Raton, Florida 33433, USA
    Crit Rev Oncog 18:177-95. 2013
  7. pmc Reducing selection bias in case-control studies from rare disease registries
    J Alexander Cole
    Biomedical Data Sciences and Informatics, Genzyme, a Sanofi Company, 500 Kendall Street, Cambridge, MA 02142, USA
    Orphanet J Rare Dis 6:61. 2011
  8. doi request reprint A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase
    Neal Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Northwest Oncology Hematology Associates PA, Coral Springs, Florida 33065, USA
    Am J Hematol 83:890-5. 2008
  9. ncbi request reprint Imiglucerase and its use for the treatment of Gaucher's disease
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Inc, Northwest Oncology Hematology Associates PA, 8170 Royal Palm Boulevard, Coral Springs, FL 33065, USA
    Expert Opin Pharmacother 9:1987-2000. 2008
  10. ncbi request reprint Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry
    Neal J Weinreb
    Department of Medicine, University Hospital, Tamarac, Florida 33321, USA
    Am J Med 113:112-9. 2002

Detail Information

Publications18

  1. ncbi request reprint Imiglucerase (Cerezyme) improves quality of life in patients with skeletal manifestations of Gaucher disease
    N Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Coral Springs, FL 33065, USA
    Clin Genet 71:576-88. 2007
    ..Imiglucerase treatment has a significant positive impact on HRQOL of type 1 GD patients with skeletal disease, including those with bone infarctions, lytic lesions, and avascular necrosis...
  2. doi request reprint A validated disease severity scoring system for adults with type 1 Gaucher disease
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Disorders, Coral Springs, FL 33065, USA
    Genet Med 12:44-51. 2010
    ..A validated disease severity scoring system (DS3) for Gaucher disease type 1 (GD1) is needed to standardize patient monitoring and to define patient cohorts in clinical studies...
  3. ncbi request reprint Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes
    Marissa Orenstein
    Dr John T, Macdonald Foundation Department of Human Genetics, Miller School of Medicine, Miami, FL, USA
    Orphanet J Rare Dis 9:45. 2014
    ..The ethnically diverse, largely immigrant population in South Florida has a spectrum of Gaucher disease phenotypes, creating a challenge for optimization of disease management and an opportunity to explore treatment patterns...
  4. ncbi request reprint Oral small molecule therapy for lysosomal storage diseases
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Boca Raton, Florida 33433, USA
    Pediatr Endocrinol Rev 11:77-90. 2013
    ..This article reviews current knowledge of small molecule treatment for LSDs including approaches such as substrate synthesis inhibition, pharmacological chaperones, and proteostasis modification...
  5. pmc Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Inc, Northwest Oncology Hematology Associates PA, Coral Springs, FL 33065, USA
    J Inherit Metab Dis 36:543-53. 2013
    ..We studied the effect of long-term alglucerase/imiglucerase (Ceredase®/Cerezyme®, Genzyme, a Sanofi company, Cambridge, MA, USA) treatment on hematological, visceral, and bone manifestations of Gaucher disease type 1 (GD1)...
  6. ncbi request reprint Causes of death due to hematological and non-hematological cancers in 57 US patients with type 1 Gaucher Disease who were never treated with enzyme replacement therapy
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Boca Raton, Florida 33433, USA
    Crit Rev Oncog 18:177-95. 2013
    ....
  7. pmc Reducing selection bias in case-control studies from rare disease registries
    J Alexander Cole
    Biomedical Data Sciences and Informatics, Genzyme, a Sanofi Company, 500 Kendall Street, Cambridge, MA 02142, USA
    Orphanet J Rare Dis 6:61. 2011
    ..Data from the International Collaborative Gaucher Group (ICGG) Gaucher Registry were used as an example...
  8. doi request reprint A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase
    Neal Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Northwest Oncology Hematology Associates PA, Coral Springs, Florida 33065, USA
    Am J Hematol 83:890-5. 2008
    ..This analysis provides a benchmark for evaluating the utility of a disease management approach for GD1 based on monitoring achievement of therapeutic goals after treatment with imiglucerase...
  9. ncbi request reprint Imiglucerase and its use for the treatment of Gaucher's disease
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Inc, Northwest Oncology Hematology Associates PA, 8170 Royal Palm Boulevard, Coral Springs, FL 33065, USA
    Expert Opin Pharmacother 9:1987-2000. 2008
    ....
  10. ncbi request reprint Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry
    Neal J Weinreb
    Department of Medicine, University Hospital, Tamarac, Florida 33321, USA
    Am J Med 113:112-9. 2002
    ..Previous studies in relatively small numbers of patients demonstrated short-term efficacy of this treatment. This study describes the effects of 2 to 5 years of treatment on specific manifestations of type 1 Gaucher disease...
  11. ncbi request reprint Guidance on the use of miglustat for treating patients with type 1 Gaucher disease
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases and Northwest Oncology Hematology Associates PA, Coral Springs, Florida
    Am J Hematol 80:223-9. 2005
    ....
  12. ncbi request reprint Individualization of long-term enzyme replacement therapy for Gaucher disease
    Hans C Andersson
    Human Genetics Program, Hayward Genetics Center, Tulane University Medical School, New Orleans, Louisiana, USA
    Genet Med 7:105-10. 2005
    ..In this review, we present recommendations for initial imiglucerase treatment and subsequent dose adjustments based on a schedule of regular assessment and monitoring, and achievement and maintenance of defined therapeutic goals...
  13. ncbi request reprint Therapeutic goals in the treatment of Gaucher disease
    Gregory M Pastores
    Neurology in Pediatrics, Neurgenetics Unit, Department of Neurology, New York University School of Medicine, NY, USA
    Semin Hematol 41:4-14. 2004
    ..Here we establish goals of treatment in Gaucher disease and propose a comprehensive schedule of monitoring of all relevant aspects to confirm the achievement, maintenance, and continuity of the therapeutic response...
  14. ncbi request reprint Gaucher disease and cancer incidence: a study from the Gaucher Registry
    Barry E Rosenbloom
    Tower Hematology Oncology Medical Group, Beverly Hills, CA 90211, USA
    Blood 105:4569-72. 2005
    ..However, there appears to be a significantly higher risk of multiple myeloma of which physicians should be aware when caring for these patients...
  15. ncbi request reprint Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients
    Neal J Weinreb
    University Research Foundation for Lysosomal Storage Diseases, Department of Medicine, University Hospital, Tamarac, FL, USA
    Semin Hematol 41:15-22. 2004
    ..Additionally, reassessment should be performed whenever enzyme therapy dose is altered, or in case of significant clinical complication...
  16. ncbi request reprint Introduction. Advances in Gaucher Disease: therapeutic goals and evaluation and monitoring guidelines
    Neal J Weinreb
    International Collaborative Gaucher Group, University Research Foundation for Lysosomal Storage Diseases, Inc, Coral Springs, FL, USA
    Semin Hematol 41:1-3. 2004
  17. ncbi request reprint Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations
    Joel Charrow
    Division of Genetics, Children s Memorial Hospital and the Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois 60614, USA
    J Pediatr 144:112-20. 2004
  18. doi request reprint Prevalence of type 1 Gaucher disease in the United States
    Neal J Weinreb
    Arch Intern Med 168:326-7; author reply 327-8. 2008