Financial support
The study was co-funded equally by Biomarin, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Shire, Solid Biosciences, and Catabasis Therapeutics. Susan J. Ward received funding from Cure Duchenne to co-develop the idea for the study and was supported with funds from Cure Duchenne and Parent Project Muscular Dystrophy to establish the cTAP. Analysis Group Inc. was a pro bono partner in establishing cTAP. Collection of the natural history data was funded by the Fondazione Telethon