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Neurology
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December 1, 2022
Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses
Kyriakos Martakis, Jens Claassen, Jordi Gascon-Bayari, et al.
International Journal of Rheumatic Diseases
|
July 16, 2015
A prospective, randomized, double-blind, multicentre, parallel-group, active controlled study to compare efficacy and safety of biosimilar adalimumab (Exemptia; ZRC-3197) and adalimumab (Humira) in patients with rheumatoid arthritis
Rajendrakumar H Jani, Rajiv Gupta, Girish Bhatia, et al.
NPJ Genomic Medicine
|
December 30, 2025
Author Correction: PHKA1-associated phosphorylase kinase deficiency: a monogenic disorder of exercise intolerance and myalgia
Rebecca L Koch, Angie H Fares, Benjamin T Cocanougher, et al.
Drug Design, Development and Therapy
|
July 18, 2015
Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
Lubor Goláň, Ozlem Goker-Alpan, Myrl Holida, et al.
Journal of Neurology
|
August 13, 2021
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C
Tatiana Bremova-Ertl, Jens Claassen, Tomas Foltan, et al.
International Journal of Rheumatic Diseases
|
June 2, 2020
Tofacitinib in the treatment of Indian patients with rheumatoid arthritis: A post hoc analysis of efficacy and safety in Phase 3 and long-term extension studies over 7 years
Arvind Chopra, Vineeta Shobha, Srikantiah Chandrashekara, et al.
Hepatology (Baltimore, Md.)
|
January 26, 2013
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease
Manisha Balwani, Catherine Breen, Gregory M Enns, et al.
Orphanet Journal of Rare Diseases
|
October 15, 2021
In-depth phenotyping for clinical stratification of Gaucher disease
Simona D'Amore, Kathleen Page, Aimée Donald, et al.
Orphanet Journal of Rare Diseases
|
February 15, 2022
Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR)
Shaun C Bolton, Vina Soran, Mercedes Pineda Marfa, et al.
Molecular Genetics and Metabolism
|
July 15, 2025
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial
Eugen Mengel, Rosalia M Da Riol, Mireia Del Toro, et al.
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of 15
Search research articles
Search
Showing results (131-140 of 146) with videos related to
Sort By:
Page
of 15
Neurology
|
December 1, 2022
Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses
Kyriakos Martakis, Jens Claassen, Jordi Gascon-Bayari, et al.
International Journal of Rheumatic Diseases
|
July 16, 2015
A prospective, randomized, double-blind, multicentre, parallel-group, active controlled study to compare efficacy and safety of biosimilar adalimumab (Exemptia; ZRC-3197) and adalimumab (Humira) in patients with rheumatoid arthritis
Rajendrakumar H Jani, Rajiv Gupta, Girish Bhatia, et al.
NPJ Genomic Medicine
|
December 30, 2025
Author Correction: PHKA1-associated phosphorylase kinase deficiency: a monogenic disorder of exercise intolerance and myalgia
Rebecca L Koch, Angie H Fares, Benjamin T Cocanougher, et al.
Drug Design, Development and Therapy
|
July 18, 2015
Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
Lubor Goláň, Ozlem Goker-Alpan, Myrl Holida, et al.
Journal of Neurology
|
August 13, 2021
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C
Tatiana Bremova-Ertl, Jens Claassen, Tomas Foltan, et al.
International Journal of Rheumatic Diseases
|
June 2, 2020
Tofacitinib in the treatment of Indian patients with rheumatoid arthritis: A post hoc analysis of efficacy and safety in Phase 3 and long-term extension studies over 7 years
Arvind Chopra, Vineeta Shobha, Srikantiah Chandrashekara, et al.
Hepatology (Baltimore, Md.)
|
January 26, 2013
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease
Manisha Balwani, Catherine Breen, Gregory M Enns, et al.
Orphanet Journal of Rare Diseases
|
October 15, 2021
In-depth phenotyping for clinical stratification of Gaucher disease
Simona D'Amore, Kathleen Page, Aimée Donald, et al.
Orphanet Journal of Rare Diseases
|
February 15, 2022
Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR)
Shaun C Bolton, Vina Soran, Mercedes Pineda Marfa, et al.
Molecular Genetics and Metabolism
|
July 15, 2025
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial
Eugen Mengel, Rosalia M Da Riol, Mireia Del Toro, et al.
Page
of 15