Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy

Published:2024-03-12 Issue:5 Volume:102 Page:
ISSN:0028-3878
Container-title:Neurology
language:en
Short-container-title:Neurology

Author:

Dang Utkarsh J.¹,Damsker Jesse M.¹^ORCID,Guglieri Michela¹^ORCID,Clemens Paula R.¹,Perlman Seth J.¹,Smith Edward C.¹,Horrocks Iain¹,Finkel Richard S.¹^ORCID,Mah Jean K.¹^ORCID,Deconinck Nicolas¹,Goemans Nathalie M.¹^ORCID,Haberlová Jana¹,Straub Volker¹^ORCID,Mengle-Gaw Laurel¹,Schwartz Benjamin D.¹,Harper Amy¹,Shieh Perry B.¹,De Waele Liesbeth¹,Castro Diana¹,Yang Michele L.¹,Ryan Monique M.¹^ORCID,McDonald Craig M.¹^ORCID,Tulinius Mar¹,Webster Richard I.¹,Mcmillan Hugh J.¹^ORCID,Kuntz Nancy¹,Rao Vamshi K.¹,Baranello Giovanni¹,Spinty Stefan¹^ORCID,Childs Anne-Marie¹,Sbrocchi Annie M.¹,Selby Kathryn A.¹,Monduy Migvis¹,Nevo Yoram¹,Vilchez Juan J.¹^ORCID,Nascimento-Osorio Andres¹,Niks Erik H.¹^ORCID,De Groot Imelda J.M.¹,Katsalouli Marina¹,Van Den Anker John N.¹,Ward Leanne M.¹^ORCID,Leinonen Mika¹,D'Alessandro Andrea L.¹,Hoffman Eric P.¹^ORCID

Affiliation:

1. From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal...

Publisher

Ovid Technologies (Wolters Kluwer Health)

Link

https://www.neurology.org/doi/pdfdirect/10.1212/WNL.0000000000208112

Reference25 articles.

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2. Dystrophin: The protein product of the duchenne muscular dystrophy locus

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4. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

5. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

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1. Corticosteroids for the treatment of Duchenne muscular dystrophy: a safety review;Expert Opinion on Drug Safety;2024-08-27

2. Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis;Frontiers in Neurology;2024-08-21

3. Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial;The Journal of Clinical Endocrinology & Metabolism;2024-08-04

4. Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy;2024-06-16

5. The glucocorticoid receptor acts locally to protect dystrophic muscle and heart during disease;Disease Models & Mechanisms;2024-05-01