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Plos One
|
November 15, 2023
Transcultural adaptation and validation of the Serbian version of Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-Patient Satisfaction (FACIT-TS-PS) questionnaire
Ivana Radovic, Igor Krdzic, Ana Jovanovic, et al.
Molecular Genetics and Metabolism Reports
|
July 24, 2025
Iron metabolism and hematological abnormalities in adult patients affected with mucopolysaccharidoses
Gabija Kaciulyte, Goknur Yorulmaz, Reena Sharma, et al.
JIMD Reports
|
December 8, 2017
Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease
Karolina M Stepien, Su Han Lum, J Edmond Wraith, et al.
Nanoscale
|
May 11, 2023
Defect-free graphene enhances enzyme delivery to fibroblasts derived from patients with lysosomal storage disorders
Yingxian Chen, Tooba Taufiq, Niting Zeng, et al.
Orphanet Journal of Rare Diseases
|
January 7, 2022
Cardiac device implantation and device usage in Fabry and hypertrophic cardiomyopathy
Ravi Vijapurapu, William Bradlow, Francisco Leyva, et al.
JIMD Reports
|
May 17, 2017
Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry
William R Wilcox, Ulla Feldt-Rasmussen, Ana Maria Martins, et al.
Frontiers in Cardiovascular Medicine
|
December 25, 2023
Clinical utilisation of implantable loop recorders in adults with Fabry disease-a multi-centre snapshot study
Ashwin Roy, Ravi Vijapurapu, Hibba Kurdi, et al.
Orphanet Journal of Rare Diseases
|
October 21, 2023
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study
Aleš Linhart, Gabriela Dostálová, Kathy Nicholls, et al.
Molecular Genetics and Metabolism Reports
|
November 9, 2020
Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype
Robert J Hopkin, Ulla Feldt-Rasmussen, Dominique P Germain, et al.
American Journal of Medical Genetics. Part A
|
March 29, 2019
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study
Derralynn A Hughes, Kathleen Nicholls, Gere Sunder-Plassmann, et al.
Page
of 6
Search research articles
Search
Showing results (31-40 of 57) with videos related to
Sort By:
Page
of 6
Plos One
|
November 15, 2023
Transcultural adaptation and validation of the Serbian version of Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-Patient Satisfaction (FACIT-TS-PS) questionnaire
Ivana Radovic, Igor Krdzic, Ana Jovanovic, et al.
Molecular Genetics and Metabolism Reports
|
July 24, 2025
Iron metabolism and hematological abnormalities in adult patients affected with mucopolysaccharidoses
Gabija Kaciulyte, Goknur Yorulmaz, Reena Sharma, et al.
JIMD Reports
|
December 8, 2017
Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease
Karolina M Stepien, Su Han Lum, J Edmond Wraith, et al.
Nanoscale
|
May 11, 2023
Defect-free graphene enhances enzyme delivery to fibroblasts derived from patients with lysosomal storage disorders
Yingxian Chen, Tooba Taufiq, Niting Zeng, et al.
Orphanet Journal of Rare Diseases
|
January 7, 2022
Cardiac device implantation and device usage in Fabry and hypertrophic cardiomyopathy
Ravi Vijapurapu, William Bradlow, Francisco Leyva, et al.
JIMD Reports
|
May 17, 2017
Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry
William R Wilcox, Ulla Feldt-Rasmussen, Ana Maria Martins, et al.
Frontiers in Cardiovascular Medicine
|
December 25, 2023
Clinical utilisation of implantable loop recorders in adults with Fabry disease-a multi-centre snapshot study
Ashwin Roy, Ravi Vijapurapu, Hibba Kurdi, et al.
Orphanet Journal of Rare Diseases
|
October 21, 2023
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study
Aleš Linhart, Gabriela Dostálová, Kathy Nicholls, et al.
Molecular Genetics and Metabolism Reports
|
November 9, 2020
Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype
Robert J Hopkin, Ulla Feldt-Rasmussen, Dominique P Germain, et al.
American Journal of Medical Genetics. Part A
|
March 29, 2019
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study
Derralynn A Hughes, Kathleen Nicholls, Gere Sunder-Plassmann, et al.
Page
of 6