Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids
Nathalie Goemans; M. Tulinius; Anna‐Karin Kroksmark; Rosamund Wilson; M. van den Hauwe; Giles Campion · 2016 · Neuromuscular Disorders
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract (excerpt)
Duchenne muscular dystrophy is a rare genetic disorder with life-limiting pathology. Drisapersen induces exon 51 skipping, thereby producing a shorter but functional dystrophin protein. The longest available data are from an open-label…
Excerpt shown for reference under fair use — read the full paper at the publisher.
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Metadata source: OpenAlex · DOI 10.1016/j.nmd.2016.11.013