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American Journal of Hematology
|
January 23, 2013
Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase
Ari Zimran, Gregory M Pastores, Anna Tylki-Szymanska, et al.
Plos One
|
July 5, 2013
Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT
Suhrad G Banugaria, Sean N Prater, Trusha T Patel, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 25, 2013
Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry
Dominique P Germain, Frank Weidemann, Ademola Abiose, et al.
Neuro-Oncology
|
April 5, 2012
Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysis
Michael J Fisher, Michael Loguidice, David H Gutmann, et al.
The New England Journal of Medicine
|
July 4, 2019
C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia
Ravi Savarirayan, Melita Irving, Carlos A Bacino, et al.
American Journal of Medical Genetics. Part C, Seminars in Medical Genetics
|
July 18, 2024
Expanding the phenotype of neurofibromatosis type 1 microdeletion syndrome
Jenny P Garzon, Andrea Patete, Lindsey Aschbacher-Smith, et al.
Annals of Neurology
|
April 18, 2014
Gender as a disease modifier in neurofibromatosis type 1 optic pathway glioma
Michael J Fisher, Michael Loguidice, David H Gutmann, et al.
The Lancet. Child & Adolescent Health
|
November 20, 2023
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial
Ravi Savarirayan, William R Wilcox, Paul Harmatz, et al.
Blood Cells, Molecules & Diseases
|
April 28, 2019
Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Blood Cells, Molecules & Diseases
|
April 23, 2018
Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
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of 11
Search research articles
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Showing results (91-100 of 110) with videos related to
Sort By:
Page
of 11
American Journal of Hematology
|
January 23, 2013
Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase
Ari Zimran, Gregory M Pastores, Anna Tylki-Szymanska, et al.
Plos One
|
July 5, 2013
Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT
Suhrad G Banugaria, Sean N Prater, Trusha T Patel, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 25, 2013
Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry
Dominique P Germain, Frank Weidemann, Ademola Abiose, et al.
Neuro-Oncology
|
April 5, 2012
Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysis
Michael J Fisher, Michael Loguidice, David H Gutmann, et al.
The New England Journal of Medicine
|
July 4, 2019
C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia
Ravi Savarirayan, Melita Irving, Carlos A Bacino, et al.
American Journal of Medical Genetics. Part C, Seminars in Medical Genetics
|
July 18, 2024
Expanding the phenotype of neurofibromatosis type 1 microdeletion syndrome
Jenny P Garzon, Andrea Patete, Lindsey Aschbacher-Smith, et al.
Annals of Neurology
|
April 18, 2014
Gender as a disease modifier in neurofibromatosis type 1 optic pathway glioma
Michael J Fisher, Michael Loguidice, David H Gutmann, et al.
The Lancet. Child & Adolescent Health
|
November 20, 2023
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial
Ravi Savarirayan, William R Wilcox, Paul Harmatz, et al.
Blood Cells, Molecules & Diseases
|
April 28, 2019
Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Blood Cells, Molecules & Diseases
|
April 23, 2018
Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Page
of 11