Journal article

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

Laurie S Conklin, Jesse M Damsker, Eric P Hoffman, William J Jusko, Panteleimon D Mavroudis, Benjamin D Schwartz, Laurel J Mengle-Gaw, Edward C Smith, Jean K Mah, Michela Guglieri, Yoram Nevo, Nancy Kuntz, Craig M McDonald, Mar Tulinius, Monique M Ryan, Richard Webster, Diana Castro, Richard S Finkel, Andrea L Smith, Lauren P Morgenroth Show all

Pharmacological Research | ACADEMIC PRESS LTD- ELSEVIER SCIENCE LTD | Published : 2018

Grants

Awarded by National Institutes of Health grant from the National Institute for Neurological Diseases and Stroke


Awarded by National Institutes of Health grant from the National Institute for Arthritis and Musculoskeletal and Skin Diseases


Awarded by National Institutes of Health grant from the Eunice Kennedy Shriver National Institute of Child Health and Human Development


Awarded by Department of Defense


Awarded by Muscular Dystrophy Association


Funding Acknowledgements

This work was funded by a National Institutes of Health grant from the National Institute for Neurological Diseases and Stroke, (R44NS095423) (EPH, PRC), a National Institutes of Health grant from the National Institute for Arthritis and Musculoskeletal and Skin Diseases (1U34AR068616) (PRC), a National Institutes of Health grant from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (5U54HD090254) (JvdA, EPH, LSC, YH), a Department of Defense grant W81XWH-15-1-0265 (YH), and a Muscular Dystrophy Association grant MDA353094 (YH). Support for the vamorolone development program was provided by the Muscular Dystrophy Association (USA), Foundation to Eradicate Duchenne (USA), Parent Project Muscular Dystrophy (USA), DuchenneUK [UK], Joining Jack [UK], Duchenne Children's Trust [UK], Duchenne Research Fund (UK), Save Our Sons (Aus), Michael's Cause (USA), Pietro's Fight (USA), Alex's Wish (UK), Ryan's Quest (USA), CureDuchenne (USA). Vamorolone was developed through a partner. ship with the National Institutes of Health NCATS TRND program (Therapeutics for Rare and Neglected Disease), with support for drug production, formulation, and toxicology studies.