Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy
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Published:2022-01-25
Issue:1
Volume:5
Page:e2144178
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ISSN:2574-3805
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Container-title:JAMA Network Open
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language:en
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Short-container-title:JAMA Netw Open
Author:
Mah Jean K.1, Clemens Paula R.2, Guglieri Michela3, Smith Edward C.4, Finkel Richard S.56, Tulinius Mar7, Nevo Yoram8, Ryan Monique M.9, Webster Richard10, Castro Diana11, Kuntz Nancy L.12, McDonald Craig M.13, Damsker Jesse M.14, Schwartz Benjamin D.15, Mengle-Gaw Laurel J.15, Jackowski Stefan1617, Stimpson Georgia18, Ridout Deborah A.18, Ayyar-Gupta Vandana18, Baranello Giovanni18, Manzur Adnan Y.18, Muntoni Francesco18, Gordish-Dressman Heather19, Leinonen Mika20, Ward Leanne M.16, Hoffman Eric P.1421, Dang Utkarsh J.2122, Muntoni Francesco23, Manzur Adnan23, Baranello Giovanni23, Robb Stephanie23, Quinlivan Ros23, Sarkozy Anna23, Munot Pinki23, Main Marion23, Abbot Lianne23, Straub Volker23, Guglieri Michela23, Bertolli Chiara23, Mayhew Anna23, Muni-Lofra Robert23, James Meredith23, Sodhi Jassi23, Parasuraman Deepak23, Alhaswani Zoya23, McMurchie Heather23, Rabb Rosanna23, Childs Anne-Marie23, Pysden Karen23, Pallant Lindsey23, Small Tiffany23, Spinty Stefan23, Madhu Rajesh23, Shillington Alison23, Gregson Sarah23, Wraige Elizabeth23, Jungbluth Heinz23, Gowda Vasantha23, Sheehan Jennie23, Hughes Imelda23, Warner Sinead23, Davies Emily23, Willis Tracey23, Kulshrestha Richa23, Emery Nicholas23, Strachan Kate23, Ong Min23, White Kay23, Skone Kate23, Gibbon Frances23, Parsons Bethan23, Majumdar Anirban23, Vijaykumar Kayal23, Mason Faye23, Frimpong-Ansah Claire23, Naismith Karen23, Burslem Julie23, Horrocks Iain23, Di Marco Marina23, Brown Sarah23, Williamson Sarah23, Spencer Kirstie23, Chow Gabby23, de Goede Christian23, Selley Andrea23, Thomas Neil23, Illingworth Marjorie23, Greary Michelle23, Palmer Jenni23, White Cathy23, Greenfield Kate23, Nic Fhirleinn Grainne23, Douglas Melanie23, Tiraputhi Sandya23, Hussain Nahin23, Julien Yvonne23, Ambegaonkar Gautam23, Krishnakumar Deepa23, Taylor Jacqui23, Tewnion Jane23, Stephens Elma23, Chandratre Saleel23, Ramdas Sithara23, Ramjattan Hayley23, Baxter Alex23, Eadie Clare23, McDonald Craig M23, Henricson Erik K23, Abresch R Ted23, Joyce Nanette C23, Viswanathan Venkatarman23, Chidambaranathan Sivaprakasam23, Biggar Douglas23, McAdam Laura C23, Mah Jean K23, Tulinius Mar23, Cnaan Avital23, Morgenroth Lauren P23, Leshner Robert23, Tesi-Rocha Carolina23, Thangarajh Mathula23, Duong Tina23, Kornberg Andrew23, Ryan Monique23, Nevo Yoram23, Dubrovsky Alberto23, Clemens Paula R23, Abdel-Hamid Hoda23, Connolly Anne M23, Pestronk Alan23, Teasley Jean23, Bertorini Tulio E23, Webster Richard23, Kolski Hanna23, Kuntz Nancy23, Driscoll Sherilyn W23, Bodensteiner John B23, Carlo Jose23, Gorni Ksenija23, Lotze Timothy23, Day John W23, Karachunski Peter23,
Affiliation:
1. Alberta Children’s Hospital Research Institute, University of Calgary, Calgary, Canada 2. University of Pittsburgh and Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania 3. John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom 4. Duke University Medical Center, Durham, North Carolina 5. Nemours Children’s Hospital, Orlando, Florida 6. St. Jude Children’s Research Hospital, Memphis, Tennessee 7. Queen Silvia Children’s Hospital, Gothenburg, Sweden 8. Schneider Children’s Medical Center of Israel, Tel Aviv University, Petah Tikvah, Israel 9. Royal Children’s Hospital and Murdoch Children’s Research Institute, Melbourne, Australia 10. The Children’s Hospital at Westmead, Sydney, Australia 11. University of Texas Southwestern, Dallas 12. Ann and Robert H. Lurie Children’s Hospital, Chicago, Illinois 13. University of California Davis, Sacramento 14. ReveraGen BioPharma, Rockville, Maryland 15. Camden Group, St Louis, Missouri 16. Children’s Hospital of Eastern Ontario, Ottawa, Ontario, Canada 17. University of Saskatchewan, Saskatoon, Canada 18. Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom 19. Children’s National Hospital, Washington, District of Columbia 20. Santhera Pharmaceuticals, Pratteln, Switzerland 21. Binghamton University-State University of New York, Binghamton 22. Carleton University, Ottawa, Canada 23. for the NorthStar UK Network and CINRG DNHS Investigators
Publisher
American Medical Association (AMA)
Cited by
46 articles.
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