Abstract
Loss of ability to rise from supine is an important early clinical milestone in DMD. We investigated prognostic factors for time to loss of rise ability in 316 ambulatory boys (median age = 7.9 years, range 4.4-19.4 years; 64% on deflazacort, 36% on prednisone) with DMD drawn from four natural history databases (UZ Leuven, PRO-DMD-01 data provided by CureDuchenne, iMDEX, and ImagingDMD). Patients’ inability to rise was either explicitly recorded, or inferred based on having a recorded rise from supine completion time greater than 30 seconds.
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