Search research articles
Contact Us
Filters
Showing results (51-60 of 110) with videos related to
Page
of 11
Sort By:
American Journal of Hematology
|
October 26, 2005
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease
Neal J Weinreb, John A Barranger, Joel Charrow, et al.
American Journal of Hematology
|
May 22, 2020
Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
Pramod K Mistry, Manisha Balwani, Joel Charrow, et al.
American Journal of Medical Genetics. Part A
|
February 10, 2026
Rising Inpatient Demands for Inherited Metabolic Disorders: Impact on Pediatric Capacity
Maria Paula Silva, Kirsten Havens, Katherine Arduini, et al.
Journal of Inherited Metabolic Disease
|
September 15, 2012
Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
Neal J Weinreb, Jack Goldblatt, Jacobo Villalobos, et al.
Molecular Genetics and Metabolism
|
November 24, 2023
Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease
Carly A Rasmussen, Allegra Quadri, Erika Vucko, et al.
Molecular Genetics and Metabolism
|
September 22, 2015
Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States
Manisha Balwani, Thomas Andrew Burrow, Joel Charrow, et al.
Molecular Genetics and Metabolism
|
September 12, 2016
Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease
Deborah Elstein, T Andrew Burrow, Joel Charrow, et al.
The Journal of Molecular Diagnostics : JMD
|
July 22, 2022
Utility and Outcomes of the 2019 American College of Medical Genetics and Genomics-Clinical Genome Resource Guidelines for Interpretation of Copy Number Variants with Borderline Classifications at an Academic Clinical Diagnostic Laboratory
Andy Drackley, Casey Brew, Alissa Wlodaver, et al.
Molecular Genetics and Metabolism
|
February 28, 2012
Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells
Suhrad G Banugaria, Trusha T Patel, Joanne Mackey, et al.
Journal of Medical Genetics
|
October 23, 2015
Antiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy
David G Warnock, Christie P Thomas, Bojan Vujkovac, et al.
Page
of 11
Search research articles
Search
Showing results (51-60 of 110) with videos related to
Sort By:
Page
of 11
American Journal of Hematology
|
October 26, 2005
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease
Neal J Weinreb, John A Barranger, Joel Charrow, et al.
American Journal of Hematology
|
May 22, 2020
Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
Pramod K Mistry, Manisha Balwani, Joel Charrow, et al.
American Journal of Medical Genetics. Part A
|
February 10, 2026
Rising Inpatient Demands for Inherited Metabolic Disorders: Impact on Pediatric Capacity
Maria Paula Silva, Kirsten Havens, Katherine Arduini, et al.
Journal of Inherited Metabolic Disease
|
September 15, 2012
Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
Neal J Weinreb, Jack Goldblatt, Jacobo Villalobos, et al.
Molecular Genetics and Metabolism
|
November 24, 2023
Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease
Carly A Rasmussen, Allegra Quadri, Erika Vucko, et al.
Molecular Genetics and Metabolism
|
September 22, 2015
Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States
Manisha Balwani, Thomas Andrew Burrow, Joel Charrow, et al.
Molecular Genetics and Metabolism
|
September 12, 2016
Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease
Deborah Elstein, T Andrew Burrow, Joel Charrow, et al.
The Journal of Molecular Diagnostics : JMD
|
July 22, 2022
Utility and Outcomes of the 2019 American College of Medical Genetics and Genomics-Clinical Genome Resource Guidelines for Interpretation of Copy Number Variants with Borderline Classifications at an Academic Clinical Diagnostic Laboratory
Andy Drackley, Casey Brew, Alissa Wlodaver, et al.
Molecular Genetics and Metabolism
|
February 28, 2012
Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells
Suhrad G Banugaria, Trusha T Patel, Joanne Mackey, et al.
Journal of Medical Genetics
|
October 23, 2015
Antiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy
David G Warnock, Christie P Thomas, Bojan Vujkovac, et al.
Page
of 11