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Orphanet Journal of Rare Diseases
|
March 21, 2026
Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study
Myrl Holida, Aleš Linhart, Nicola Longo, et al.
Movement Disorders : Official Journal of the Movement Disorder Society
|
December 11, 2012
The glucocerobrosidase E326K variant predisposes to Parkinson's disease, but does not cause Gaucher's disease
Raquel Duran, Niccolo E Mencacci, Aikaterini V Angeli, et al.
Journal of Inherited Metabolic Disease
|
October 9, 2024
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies
Myrl Holida, Aleš Linhart, Antonio Pisani, et al.
Blood Cells, Molecules & Diseases
|
October 7, 2009
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease
Carla E M Hollak, Stephan vom Dahl, Johannes M F G Aerts, et al.
The New England Journal of Medicine
|
September 10, 2015
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
Barbara K Burton, Manisha Balwani, François Feillet, et al.
Journal of Medical Genetics
|
November 12, 2016
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
Derralynn A Hughes, Kathleen Nicholls, Suma P Shankar, et al.
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Search research articles
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Showing results (41-50 of 46) with videos related to
Sort By:
Page
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You have reached the last page of results.
This site can display upto 46 results.
Orphanet Journal of Rare Diseases
|
March 21, 2026
Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study
Myrl Holida, Aleš Linhart, Nicola Longo, et al.
Movement Disorders : Official Journal of the Movement Disorder Society
|
December 11, 2012
The glucocerobrosidase E326K variant predisposes to Parkinson's disease, but does not cause Gaucher's disease
Raquel Duran, Niccolo E Mencacci, Aikaterini V Angeli, et al.
Journal of Inherited Metabolic Disease
|
October 9, 2024
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies
Myrl Holida, Aleš Linhart, Antonio Pisani, et al.
Blood Cells, Molecules & Diseases
|
October 7, 2009
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease
Carla E M Hollak, Stephan vom Dahl, Johannes M F G Aerts, et al.
The New England Journal of Medicine
|
September 10, 2015
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
Barbara K Burton, Manisha Balwani, François Feillet, et al.
Journal of Medical Genetics
|
November 12, 2016
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
Derralynn A Hughes, Kathleen Nicholls, Suma P Shankar, et al.
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of 5