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Steroids
|
November 20, 2025
Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children
Swati Mummidivarpu, Utkarsh J Dang, Michael Ziemba, et al.
Journal of Neurology
|
August 16, 2025
Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial
Priya S Kishnani, Jordi Díaz-Manera, Sergey Illarioshkin, et al.
Nature Medicine
|
February 5, 2025
First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy
Genís Prat-Ortega, Scott Ensel, Serena Donadio, et al.
Journal of Personalized Medicine
|
November 24, 2020
Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy
Christopher R Heier, Aiping Zhang, Nhu Y Nguyen, et al.
The New England Journal of Medicine
|
April 16, 2010
A randomized study of alglucosidase alfa in late-onset Pompe's disease
Ans T van der Ploeg, Paula R Clemens, Deyanira Corzo, et al.
Plos Medicine
|
September 21, 2020
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
Edward C Smith, Laurie S Conklin, Eric P Hoffman, et al.
JAMA Neurology
|
April 10, 2023
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial
Priya S Kishnani, Jordi Diaz-Manera, Antonio Toscano, et al.
JAMA Network Open
|
January 25, 2022
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial
Jean K Mah, Paula R Clemens, Michela Guglieri, et al.
Journal of the Endocrine Society
|
January 9, 2026
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone
Anne Marie Sbrocchi, Kathi Kinnett, Maria-Elena Lautatzis, et al.
The Lancet. Neurology
|
November 20, 2021
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial
Jordi Diaz-Manera, Priya S Kishnani, Hani Kushlaf, et al.
Page
of 40
Search research articles
Search
Showing results (381-390 of 395) with videos related to
Sort By:
Page
of 40
Steroids
|
November 20, 2025
Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children
Swati Mummidivarpu, Utkarsh J Dang, Michael Ziemba, et al.
Journal of Neurology
|
August 16, 2025
Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial
Priya S Kishnani, Jordi Díaz-Manera, Sergey Illarioshkin, et al.
Nature Medicine
|
February 5, 2025
First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy
Genís Prat-Ortega, Scott Ensel, Serena Donadio, et al.
Journal of Personalized Medicine
|
November 24, 2020
Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy
Christopher R Heier, Aiping Zhang, Nhu Y Nguyen, et al.
The New England Journal of Medicine
|
April 16, 2010
A randomized study of alglucosidase alfa in late-onset Pompe's disease
Ans T van der Ploeg, Paula R Clemens, Deyanira Corzo, et al.
Plos Medicine
|
September 21, 2020
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
Edward C Smith, Laurie S Conklin, Eric P Hoffman, et al.
JAMA Neurology
|
April 10, 2023
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial
Priya S Kishnani, Jordi Diaz-Manera, Antonio Toscano, et al.
JAMA Network Open
|
January 25, 2022
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial
Jean K Mah, Paula R Clemens, Michela Guglieri, et al.
Journal of the Endocrine Society
|
January 9, 2026
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone
Anne Marie Sbrocchi, Kathi Kinnett, Maria-Elena Lautatzis, et al.
The Lancet. Neurology
|
November 20, 2021
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial
Jordi Diaz-Manera, Priya S Kishnani, Hani Kushlaf, et al.
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of 40