List of works - Neal Weinreb

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

scientific article published in December 2008

A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability

scientific article

A new framework for evaluating the health impacts of treatment for Gaucher disease type 1.

scientific article published on 20 February 2017

A validated disease severity scoring system for adults with type 1 Gaucher disease

scientific article published on 01 January 2010

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 ⬇️

scientific article published on January 1, 2013

Causes of death in 184 patients with type 1 Gaucher disease from the United States who were never treated with enzyme replacement therapy.

scientific article published on 24 October 2016

Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years

scientific article

Early access experience with VPRIV(®): recommendations for 'core data' collection.

scientific article published on 13 December 2010

Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa

scientific article published on 31 March 2015

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

scientific article

Encore! Oral therapy for type 1 Gaucher disease

scientific article published in April 2017

Enzyme replacement and substrate reduction therapy for Gaucher disease

scientific article

Enzyme replacement and substrate reduction therapy for Gaucher disease

scientific article (published 2013-01-31)

Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations

scientific article published on January 2004

Erratum to: Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

scientific article published on 28 August 2013

Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)

scientific article published on 22 May 2015

Gaucher disease and cancer incidence: a study from the Gaucher Registry

scientific article published on 17 February 2005

Gaucher disease epidemiology and natural history: a comprehensive review of the literature.

scientific article

Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients

scientific article published on 01 October 2004

Gaucher disease: Progress and ongoing challenges

scientific article published on 17 November 2016

Gaucher disease: a comprehensive review

scientific article

Gaucher disease: resetting the clinical and scientific agenda

scientific article published on April 2009

Guidance on the use of miglustat for treating patients with type 1 Gaucher disease

scientific article

Hepatocellular carcinoma in Gaucher disease: an international case series.

scientific article published on 8 February 2018

Imiglucerase and its use for the treatment of Gaucher's disease

scientific article

Individualization of long-term enzyme replacement therapy for Gaucher disease

scientific article

Introduction. Advances in Gaucher Disease: therapeutic goals and evaluation and monitoring guidelines

scientific article published in October 2004

JAK2V617F mutation and myeloproliferative malignancy in a patient with Type 1 Gaucher disease ⬇️

scientific article published on October 23, 2010

Life expectancy in Gaucher disease type 1

scientific article

Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

scientific article

MGUS, lymphoplasmacytic malignancies and Gaucher disease: the significance of the clinical association.

scientific article published on 12 April 2018

Oral small molecule therapy for lysosomal storage diseases

scientific article

Osteopenia in Gaucher disease develops early in life: Response to imiglucerase enzyme therapy in children, adolescents and adults ⬇️

scientific article published on November 26, 2010

Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes

scientific article

Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Early Diagnosis Consensus (GED-C) Delphi initiative

scientific article published on 01 May 2019

Prevalence of type 1 Gaucher disease in the United States

scientific article published on 01 February 2008

Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States

scientific article

Reducing selection bias in case-control studies from rare disease registries ⬇️

scientific article published on September 12, 2011

Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease

scientific article published on 25 August 2015

Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease: A study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry ⬇️

scientific article published on August 1, 2012

Roscoe Owen Brady, MD: Remembrances of co-investigators and colleagues

scientific article published on 12 November 2016

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

scientific article published on 20 March 2015

The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease

scientific article published on 27 August 2014

The history and accomplishments of the ICGG Gaucher registry

scientific article published on July 2015

The incidence of Parkinsonism in patients with type 1 Gaucher disease: data from the ICGG Gaucher Registry

scientific article

The need for disease-specific patient-reported outcome measures for lysosomal disorders

Therapeutic goals in the treatment of Gaucher disease.

scientific article

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis

scientific article published on 03 September 2009

Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

scientific article published on 31 May 2017

List of works by Neal Weinreb

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability

A new framework for evaluating the health impacts of treatment for Gaucher disease type 1.

A validated disease severity scoring system for adults with type 1 Gaucher disease

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 ⬇️

Causes of death in 184 patients with type 1 Gaucher disease from the United States who were never treated with enzyme replacement therapy.

Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years

Early access experience with VPRIV(®): recommendations for 'core data' collection.

Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa

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

Encore! Oral therapy for type 1 Gaucher disease

Enzyme replacement and substrate reduction therapy for Gaucher disease

Enzyme replacement and substrate reduction therapy for Gaucher disease

Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations

Erratum to: Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)

Gaucher disease and cancer incidence: a study from the Gaucher Registry

Gaucher disease epidemiology and natural history: a comprehensive review of the literature.

Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients

Gaucher disease: Progress and ongoing challenges

Gaucher disease: a comprehensive review

Gaucher disease: resetting the clinical and scientific agenda

Guidance on the use of miglustat for treating patients with type 1 Gaucher disease

Hepatocellular carcinoma in Gaucher disease: an international case series.

Imiglucerase and its use for the treatment of Gaucher's disease

Individualization of long-term enzyme replacement therapy for Gaucher disease

Introduction. Advances in Gaucher Disease: therapeutic goals and evaluation and monitoring guidelines

JAK2V617F mutation and myeloproliferative malignancy in a patient with Type 1 Gaucher disease ⬇️

Life expectancy in Gaucher disease type 1

Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

MGUS, lymphoplasmacytic malignancies and Gaucher disease: the significance of the clinical association.

Oral small molecule therapy for lysosomal storage diseases

Osteopenia in Gaucher disease develops early in life: Response to imiglucerase enzyme therapy in children, adolescents and adults ⬇️

Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes

Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Early Diagnosis Consensus (GED-C) Delphi initiative

Prevalence of type 1 Gaucher disease in the United States

Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States

Reducing selection bias in case-control studies from rare disease registries ⬇️

Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease

Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease: A study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry ⬇️

Roscoe Owen Brady, MD: Remembrances of co-investigators and colleagues

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease

The history and accomplishments of the ICGG Gaucher registry

The incidence of Parkinsonism in patients with type 1 Gaucher disease: data from the ICGG Gaucher Registry

The need for disease-specific patient-reported outcome measures for lysosomal disorders

Therapeutic goals in the treatment of Gaucher disease.

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis

Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.