Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

Duchenne muscular dystrophy (DMD) is a progressive, lethal neuromuscular disorder caused by the absence of dystrophin protein due to mutations of the dystrophin gene. Drisapersen is a 2'- O-methyl-phosphorothioate oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA to restore the reading frame of the mRNA. This study assessed safety, tolerability, and pharmacokinetics of drisapersen after a single subcutaneous administration in non-ambulatory subjects. Eligible subjects were non-ambulant boys aged ≥9. years, in wheelchairs for ≥1 to ≤4. years, with a diagnosis of DMD resulting from a mutation correctable by drisapersen treatment. Four dose cohorts were planned (3, 6, 9 and 12. mg/kg), but study objectives were met with the 9. mg/kg dose. Less than proportional increase in exposure was demonstrated over the 3-9. mg/kg dose range, though post hoc analysis showed dose proportionality was more feasible over the 3-6. mg/kg range. Single doses of drisapersen at 3 and 6. mg/kg did not result in significant safety or tolerability concerns; however, at the 9. mg/kg dose, pyrexia and transient elevations in inflammatory parameters were seen. The maximum tolerated dose of 6. mg/kg drisapersen was identified for further characterization in multiple dose studies in the non-ambulant DMD population. © 2013 The Authors.

Original publication




Journal article


Neuromuscular Disorders

Publication Date





16 - 24