Search research articles
Contact Us
Filters
Showing results (91-100 of 117) with videos related to
Page
of 12
Sort By:
Blood
|
February 26, 2004
Spectrum of hemojuvelin gene mutations in 1q-linked juvenile hemochromatosis
Carmela Lanzara, Antonella Roetto, Filomena Daraio, et al.
Haematologica
|
July 10, 2012
Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes
Laura van Dussen, Timothy M Cox, Erik J Hendriks, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 18, 2025
Venglustat in GM2 gangliosidoses and related disorders: Results of the AMETHIST randomized controlled and basket trials
Cynthia J Tifft, Isabela Batsu, Roberto Giugliani, et al.
European Journal of Internal Medicine
|
August 15, 2016
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe
Nadia Belmatoug, Maja Di Rocco, Cristina Fraga, et al.
Brain : a Journal of Neurology
|
October 18, 2022
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
Raphael Schiffmann, Timothy M Cox, Jean-François Dedieu, et al.
Human Mutation
|
September 25, 2022
Juvenile mucopolysaccharidosis plus disease caused by a missense mutation in VPS33A
Elena V Pavlova, Dorit Lev, Marina Michelson, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 23, 2009
A validated disease severity scoring system for adults with type 1 Gaucher disease
Neal J Weinreb, Maria D Cappellini, Timothy M Cox, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
February 6, 2023
Gaucher disease protects against tuberculosis
Jingwen Fan, Victoria L Hale, Lindsey T Lelieveld, et al.
Cells
|
November 27, 2021
L-Arginine Ameliorates Defective Autophagy in GM2 Gangliosidoses by mTOR Modulation
Beatriz Castejón-Vega, Alejandro Rubio, Antonio J Pérez-Pulido, et al.
The Journal of Clinical Investigation
|
February 11, 2025
Safety and efficacy of pharmacological inhibition of ketohexokinase in hereditary fructose intolerance
Evi Jc Koene, Amée M Buziau, David Cassiman, et al.
Page
of 12
Search research articles
Search
Showing results (91-100 of 117) with videos related to
Sort By:
Page
of 12
Blood
|
February 26, 2004
Spectrum of hemojuvelin gene mutations in 1q-linked juvenile hemochromatosis
Carmela Lanzara, Antonella Roetto, Filomena Daraio, et al.
Haematologica
|
July 10, 2012
Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes
Laura van Dussen, Timothy M Cox, Erik J Hendriks, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 18, 2025
Venglustat in GM2 gangliosidoses and related disorders: Results of the AMETHIST randomized controlled and basket trials
Cynthia J Tifft, Isabela Batsu, Roberto Giugliani, et al.
European Journal of Internal Medicine
|
August 15, 2016
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe
Nadia Belmatoug, Maja Di Rocco, Cristina Fraga, et al.
Brain : a Journal of Neurology
|
October 18, 2022
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
Raphael Schiffmann, Timothy M Cox, Jean-François Dedieu, et al.
Human Mutation
|
September 25, 2022
Juvenile mucopolysaccharidosis plus disease caused by a missense mutation in VPS33A
Elena V Pavlova, Dorit Lev, Marina Michelson, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 23, 2009
A validated disease severity scoring system for adults with type 1 Gaucher disease
Neal J Weinreb, Maria D Cappellini, Timothy M Cox, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
February 6, 2023
Gaucher disease protects against tuberculosis
Jingwen Fan, Victoria L Hale, Lindsey T Lelieveld, et al.
Cells
|
November 27, 2021
L-Arginine Ameliorates Defective Autophagy in GM2 Gangliosidoses by mTOR Modulation
Beatriz Castejón-Vega, Alejandro Rubio, Antonio J Pérez-Pulido, et al.
The Journal of Clinical Investigation
|
February 11, 2025
Safety and efficacy of pharmacological inhibition of ketohexokinase in hereditary fructose intolerance
Evi Jc Koene, Amée M Buziau, David Cassiman, et al.
Page
of 12